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Sakaguchi H, Hirano D, Saito A, Takemasa Y, Umeda C, Miwa S, Ito A, Oishi K. Effectiveness and safety of Tolvaptan in infants with congenital heart disease. Pediatr Int 2023; 65:e15580. [PMID: 37428842 DOI: 10.1111/ped.15580] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Track Full Text] [Journal Information] [Submit a Manuscript] [Subscribe] [Scholar Register] [Received: 02/17/2023] [Revised: 05/20/2023] [Accepted: 05/29/2023] [Indexed: 07/12/2023]
Abstract
BACKGROUND Tolvaptan (TLV) is a selective vasopressin receptor 2 antagonist administered for congestive heart failure (CHF) after inadequate response to other diuretics. The effectiveness and safety of TLV have been evaluated well in adult patients. However, reports on its use in pediatric patients, especially infants, are scarce. METHODS We retrospectively evaluated 41 children younger than 1 year of age who received TLV for CHF for congenital heart disease (CHD) between January 2010 and August 2021. We monitored the occurrence of adverse events, including acute kidney injury and hypernatremia, as well as laboratory data trends. RESULTS Of the 41 infants included, 51.2% were male. The median age when TLV was initiated was 2 months, interquartile range (IQR) 1-4 months, and all infants had been administered other diuretics previously. The median dose of TLV was 0.1 mg/kg/day (IQR, 0.1-0.1). Urine output increased significantly after 48 h of treatment: baseline, 315 mL/day (IQR, 243-394); 48 h, 381 mL/day (IQR, 262-518) , p = 0.0004; 72 h, 385 mL/day (IQR, 301-569), p = 0.0013; 96 h, 425 mL/day (IQR, 272-524), p = 0.0006; and 144 h, 396 mL/day (IQR, 305-477), p = 0.0036. No adverse events were observed. CONCLUSIONS Tolvaptan can be used safely and efficiently in infants with CHD. From the perspective of adverse effects, initiating administration at a lower dosage is preferable because this was found to be sufficiently effective.
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Affiliation(s)
- Haruhide Sakaguchi
- Department of Pediatrics, The Jikei University School of Medicine, Tokyo, Japan
| | - Daishi Hirano
- Department of Pediatrics, The Jikei University School of Medicine, Tokyo, Japan
| | - Aya Saito
- Department of Pediatrics, The Jikei University School of Medicine, Tokyo, Japan
| | - Yoichi Takemasa
- Department of Pediatrics, The Jikei University School of Medicine, Tokyo, Japan
| | - Chisato Umeda
- Department of Pediatrics, The Jikei University School of Medicine, Tokyo, Japan
| | - Saori Miwa
- Department of Pediatrics, The Jikei University School of Medicine, Tokyo, Japan
| | - Akira Ito
- Department of Pediatrics, The Jikei University School of Medicine, Tokyo, Japan
| | - Kimihiko Oishi
- Department of Pediatrics, The Jikei University School of Medicine, Tokyo, Japan
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Meena J, Hari P, Sinha A, Bagga A. Efficacy and Safety of Combination Therapy with Tolvaptan and Furosemide in Children with Nephrotic Syndrome and Refractory Edema: A Prospective Interventional Study. Indian J Pediatr 2022; 89:699-705. [PMID: 35080742 DOI: 10.1007/s12098-021-03988-y] [Citation(s) in RCA: 1] [Impact Index Per Article: 0.3] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Journal Information] [Submit a Manuscript] [Subscribe] [Scholar Register] [Received: 05/20/2021] [Accepted: 08/19/2021] [Indexed: 11/29/2022]
Abstract
OBJECTIVE Severe edema in children with nephrotic syndrome is often refractory to conventional diuretics. Tolvaptan has been used satisfactorily for managing edema in patients with heart failure and cirrhosis. The safety and efficacy of combination therapy with oral tolvaptan and intravenous (IV) furosemide was assessed in patients with furosemide refractory edema. METHODS Patients, aged 5-18 y with nephrotic syndrome and severe edema, were screened for eligibility. After excluding hypovolemia, patients received IV furosemide (3-4 mg/kg/d) for 48 h. Those refractory to IV furosemide (weight loss < 3%) received tolvaptan (0.5-1 mg/kg once daily) and IV furosemide for the next 48 h. Parameters were compared between 48 h of furosemide alone and combination therapy. RESULTS A total of 24 patients (18 boys) with mean age of 8.0 ± 3.0 y were enrolled. Urine volume significantly increased with combination therapy as compared to furosemide therapy (mean difference: 1.2 mL/kg/h; 95% CI: 0.8-1.65 mL/kg/h) (p < 0.001). Compared to therapy with furosemide alone, combination therapy resulted in significant reduction in body weight from 26.9 ± 10.3 kg to 24.8 ± 9.7 kg (p < 0.001). Estimated glomerular filtration rate did not change (p = 0.81) but serum sodium increased from 135.7 ± 3.3 mEq/L to 140.4 ± 4.8 mEq/L (p < 0.001) with combination therapy; 2 patients showed asymptomatic hypernatremia. CONCLUSION The combination of oral tolvaptan and IV furosemide is effective in augmenting diuresis and reducing weight in patients with furosemide refractory edema but requires monitoring of electrolytes and volume status.
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Affiliation(s)
- Jitendra Meena
- Division of Nephrology, Department of Pediatrics, ICMR Center for Advanced Research in Nephrology, All India Institute of Medical Sciences, New Delhi, 110029, India
| | - Pankaj Hari
- Division of Nephrology, Department of Pediatrics, ICMR Center for Advanced Research in Nephrology, All India Institute of Medical Sciences, New Delhi, 110029, India.
| | - Aditi Sinha
- Division of Nephrology, Department of Pediatrics, ICMR Center for Advanced Research in Nephrology, All India Institute of Medical Sciences, New Delhi, 110029, India
| | - Arvind Bagga
- Division of Nephrology, Department of Pediatrics, ICMR Center for Advanced Research in Nephrology, All India Institute of Medical Sciences, New Delhi, 110029, India
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Weisert M, Su JA, Menteer J, Shaddy RE, Kantor PF. Drug Treatment of Heart Failure in Children: Gaps and Opportunities. Paediatr Drugs 2022; 24:121-136. [PMID: 35084696 DOI: 10.1007/s40272-021-00485-9] [Citation(s) in RCA: 2] [Impact Index Per Article: 0.7] [Reference Citation Analysis] [Abstract] [MESH Headings] [Track Full Text] [Journal Information] [Submit a Manuscript] [Subscribe] [Scholar Register] [Accepted: 11/08/2021] [Indexed: 12/11/2022]
Abstract
Medical therapy for pediatric heart failure is based on a detailed mechanistic understanding of the underlying causes, which are diverse and unlike those encountered in most adult patients. Diuresis and improved perfusion are the immediate goals of care in the child with acute decompensated heart failure. Conversion to maintenance oral therapy for heart failure is based on the results of landmark studies in adults, as well as recent pediatric clinical trials and heart failure guidelines. There will continue to be an important role for newer drugs, some of which are in active trials in adults, and some of which are already approved for use in children. The need to plan for clinical trials in children during drug development for heart failure is emphasized.
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Affiliation(s)
- Molly Weisert
- Department of Pediatrics, Keck School of Medicine, University of Southern California, Los Angeles, CA, USA
- Division of Cardiology, Heart Institute, Children's Hospital Los Angeles, Los Angeles, CA, USA
| | - Jennifer A Su
- Department of Pediatrics, Keck School of Medicine, University of Southern California, Los Angeles, CA, USA
- Division of Cardiology, Heart Institute, Children's Hospital Los Angeles, Los Angeles, CA, USA
| | - Jondavid Menteer
- Department of Pediatrics, Keck School of Medicine, University of Southern California, Los Angeles, CA, USA
- Division of Cardiology, Heart Institute, Children's Hospital Los Angeles, Los Angeles, CA, USA
| | - Robert E Shaddy
- Department of Pediatrics, Keck School of Medicine, University of Southern California, Los Angeles, CA, USA
- Division of Cardiology, Heart Institute, Children's Hospital Los Angeles, Los Angeles, CA, USA
| | - Paul F Kantor
- Department of Pediatrics, Keck School of Medicine, University of Southern California, Los Angeles, CA, USA.
- Division of Cardiology, Heart Institute, Children's Hospital Los Angeles, Los Angeles, CA, USA.
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Tolvaptan Response in a Hyponatremic Newborn with Syndrome of Inappropriate Secretion of Antidiuretic Hormone. Case Rep Pediatr 2021; 2021:9920817. [PMID: 34094612 PMCID: PMC8137302 DOI: 10.1155/2021/9920817] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Download PDF] [Figures] [Journal Information] [Subscribe] [Scholar Register] [Received: 03/13/2021] [Accepted: 05/07/2021] [Indexed: 11/25/2022] Open
Abstract
The use of tolvaptan to treat both euvolemic and hypervolemic hyponatremia has rapidly increased in recent years. However, data on its effects on children, especially newborns and infants, are limited. Here, we present a newborn who developed syndrome of inappropriate secretion of antidiuretic hormone following an intracranial hematoma drainage operation who was unresponsive to conventional treatments. The infant was successfully treated with tolvaptan, a competitive inhibitor of the vasopressin V2 receptor.
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Das BB, Moskowitz WB, Butler J. Current and Future Drug and Device Therapies for Pediatric Heart Failure Patients: Potential Lessons from Adult Trials. CHILDREN (BASEL, SWITZERLAND) 2021; 8:322. [PMID: 33922085 PMCID: PMC8143500 DOI: 10.3390/children8050322] [Citation(s) in RCA: 1] [Impact Index Per Article: 0.3] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Download PDF] [Figures] [Subscribe] [Scholar Register] [Received: 03/27/2021] [Revised: 04/19/2021] [Accepted: 04/20/2021] [Indexed: 12/11/2022]
Abstract
This review discusses the potential drug and device therapies for pediatric heart failure (HF) due to reduced systolic function. It is important to realize that most drugs that are used in pediatric HF are extrapolated from adult cardiology practices or consensus guidelines based on expert opinion rather than on evidence from controlled clinical trials. It is difficult to conclude whether the drugs that are well established in adult HF trials are also beneficial for children because of tremendous heterogeneity in the mechanism of HF in children and variations in the pharmacokinetics and pharmacodynamics of drugs from birth to adolescence. The lessons learned from adult trials can guide pediatric cardiologists to design clinical trials of the newer drugs that are in the pipeline to study their efficacy and safety in children with HF. This paper's focus is that the reader should specifically think through the pathophysiological mechanism of HF and the mode of action of drugs for the selection of appropriate pharmacotherapy. We review the drug and device trials in adults with HF to highlight the knowledge gap that exists in the pediatric HF population.
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Affiliation(s)
- Bibhuti B. Das
- Heart Center, Department of Pediatrics, Mississippi Children’s Hospital, University of Mississippi Medical Center, Jackson, MS 39212, USA;
| | - William B. Moskowitz
- Heart Center, Department of Pediatrics, Mississippi Children’s Hospital, University of Mississippi Medical Center, Jackson, MS 39212, USA;
| | - Javed Butler
- Department of Medicine, University of Mississippi Medical Center, Jackson, MS 39212, USA;
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Puma A, Brugnara M, Cavarzere P, Zaffanello M, Piacentini G, Gaudino R. Case Report: Long-Term Tolvaptan Treatment in a Child With SIADH and Suprasellar Arachnoid Cyst. Front Pediatr 2021; 9:684131. [PMID: 34336740 PMCID: PMC8322605 DOI: 10.3389/fped.2021.684131] [Citation(s) in RCA: 1] [Impact Index Per Article: 0.3] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Download PDF] [Figures] [Journal Information] [Submit a Manuscript] [Subscribe] [Scholar Register] [Received: 03/22/2021] [Accepted: 06/04/2021] [Indexed: 11/13/2022] Open
Abstract
Suprasellar arachnoid cysts represent a rare occurrence in the pediatric population and usually cause symptoms related to mass effect and can occasionally cause endocrine dysfunctions. The association between SAC and the syndrome of inappropriate antidiuretic hormone (SIADH) in the pediatric population has rarely been described previously. In most cases, SIADH is temporary and resolves by treating the underlying cause. The first-line treatment consists of fluid restriction in asymptomatic children. Oral urea and demeclocycline are other effective treatment options. Vaptans are a new class of medication for the management of SIADH. These agents are a nonpeptide vasopressin V2 receptor antagonist that selectively antagonizes the antidiuretic effect of AVP, resulting in excretion of diluted urine or "aquaresis." Their efficacy has been shown in adult patients with euvolemic or hypervolemic hyponatremia. However, evidence is lacking in pediatric patients with SIADH. We report the case of a 9-year-old female child with a SAC, who underwent endoscopic fenestration at the age of 2 years. After surgery she developed chronic hyponatremia due to SIADH. Hyponatremia was refractory to treatment with fluid restriction, oral sodium, and urea. In order to normalize serum sodium levels, tolvaptan treatment was started on a compassionate-use basis; 24-48 h later serum sodium levels returned to normal. To date, tolvaptan has been used regularly for 6 years with no side effects occurring during the treatment period. This is the first case of a child with chronic SIADH secondary to SAC successfully treated with tolvaptan. Further studies are needed to demonstrate its usefulness on a broader case series.
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Affiliation(s)
- Andrea Puma
- Department of Surgical Sciences, Dentistry, Gynecology and Pediatrics, Pediatric Division, University of Verona, Verona, Italy
| | - Milena Brugnara
- Department of Surgical Sciences, Dentistry, Gynecology and Pediatrics, Pediatric Nephrology Division, University of Verona, Verona, Italy
| | - Paolo Cavarzere
- Department of Surgical Sciences, Dentistry, Gynecology and Pediatrics, Pediatric Endocrinology Division, University of Verona, Verona, Italy
| | - Marco Zaffanello
- Department of Surgical Sciences, Dentistry, Gynecology and Pediatrics, Pediatric Division, University of Verona, Verona, Italy
| | - Giorgio Piacentini
- Department of Surgical Sciences, Dentistry, Gynecology and Pediatrics, Pediatric Division, University of Verona, Verona, Italy
| | - Rossella Gaudino
- Department of Surgical Sciences, Dentistry, Gynecology and Pediatrics, Pediatric Endocrinology Division, University of Verona, Verona, Italy
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Nelson CP, Kurtz MP, Venna A, Cilento BG, Baum MA. Pharmacological Dilutional Therapy Using the Vasopressin Antagonist Tolvaptan for Young Patients With Cystinuria: A Pilot Investigation. Urology 2020; 144:65-70. [DOI: 10.1016/j.urology.2020.07.002] [Citation(s) in RCA: 2] [Impact Index Per Article: 0.4] [Reference Citation Analysis] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 04/28/2020] [Revised: 06/29/2020] [Accepted: 07/05/2020] [Indexed: 12/11/2022]
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Gimpel C, Bergmann C, Brinkert F, Cetiner M, Gembruch U, Haffner D, Kemper M, König J, Liebau M, Maier RF, Oh J, Pape L, Riechardt S, Rolle U, Rossi R, Stegmann J, Vester U, Kaisenberg CV, Weber S, Schaefer F. [Kidney Cysts and Cystic Nephropathies in Children - A Consensus Guideline by 10 German Medical Societies]. KLINISCHE PADIATRIE 2020; 232:228-248. [PMID: 32659844 DOI: 10.1055/a-1179-0728] [Citation(s) in RCA: 2] [Impact Index Per Article: 0.4] [Reference Citation Analysis] [Abstract] [MESH Headings] [Track Full Text] [Subscribe] [Scholar Register] [Indexed: 02/06/2023]
Abstract
This consensus-based guideline was developed by all relevant German pediatric medical societies. Ultrasound is the standard imaging modality for pre- and postnatal kidney cysts and should also exclude extrarenal manifestations in the abdomen and internal genital organs. MRI has selected indications. Suspicion of a cystic kidney disease should prompt consultation of a pediatric nephrologist. Prenatal management must be tailored to very different degrees of disease severity. After renal oligohydramnios, we recommend delivery in a perinatal center. Neonates should not be denied renal replacement therapy solely because of their age. Children with unilateral multicystic dysplastic kidney do not require routine further imaging or nephrectomy, but long-term nephrology follow-up (as do children with uni- or bilateral kidney hypo-/dysplasia with cysts). ARPKD (autosomal recessive polycystic kidney disease), nephronophthisis, Bardet-Biedl syndrome and HNF1B mutations cause relevant extrarenal disease and genetic testing is advisable. Children with tuberous sclerosis complex, tumor predisposition (e. g. von Hippel Lindau syndrome) or high risk of acquired kidney cysts should have regular ultrasounds. Even asymptomatic children of parents with ADPKD (autosomal dominant PKD) should be monitored for hypertension and proteinuria. Presymptomatic diagnostic ultrasound or genetic examination for ADPKD in minors should only be done after thorough counselling. Simple cysts are very rare in children and ADPKD in a parent should be excluded. Complex renal cysts require further investigation.
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Affiliation(s)
- Charlotte Gimpel
- Department of Internal Medicine IV, Medical Center - University of Freiburg, Freiburg
- Faculty of Medicine, University of Freiburg, Freiburg im Breisgau
| | - Carsten Bergmann
- Department of Internal Medicine IV, Medical Center - University of Freiburg, Freiburg
- Faculty of Medicine, University of Freiburg, Freiburg im Breisgau
- Medizinische Genetik Mainz, Limbach Genetics, Mainz
| | - Florian Brinkert
- Department of Pediatrics, University Medical Center Hamburg-Eppendorf, Hamburg
| | - Metin Cetiner
- Department of Pediatrics II, University Hospital Essen, Essen
| | - Ulrich Gembruch
- Department of Obstetrics and Prenatal Medicine, University Hospital of Bonn, Bonn
| | - Dieter Haffner
- Department of Pediatric Kidney, Liver and Metabolic Diseases, Hannover Medical School, Hannover
| | - Markus Kemper
- Department of Pediatrics, Asklepios Kliniken Hamburg GmbH, Asklepios Klinik Nord, Standort Heidberg, Hamburg
| | - Jens König
- Department of General Pediatrics, University Children's Hospital Münster, Münster
| | - Max Liebau
- Department of Pediatrics, University Hospital Cologne, Cologne
- Center for Molecular Medicine, University of Cologne, Cologne
| | - Rolf Felix Maier
- Department of Pediatrics, University Hospital of Giessen and Marburg, Campus Marburg, Marburg
| | - Jun Oh
- Department of Pediatrics, University Medical Center Hamburg-Eppendorf, Hamburg
| | - Lars Pape
- Department of Pediatric Kidney, Liver and Metabolic Diseases, Hannover Medical School, Hannover
| | - Silke Riechardt
- Department of Urology, University Medical Center Hamburg-Eppendorf, Hamburg
| | - Udo Rolle
- Department of Pediatric Surgery, Hospital of the Goethe University Frankfurt, Frankfurt am Main
| | - Rainer Rossi
- Department of Pediatrics, Vivantes Klinikum Neukölln, Berlin
| | - Joachim Stegmann
- Department of Radiology, Catholic Children's Hospital Wilhelmstift, Hamburg
| | - Udo Vester
- Department of Pediatrics, HELIOS Hospital Duisburg, Duisburg
| | - Constantin von Kaisenberg
- Department of Obstetrics and Gynaecology, Center for Perinatal Medicine, Hannover Medical School, Hannover
| | - Stefanie Weber
- Department of Pediatrics, University Hospital of Giessen and Marburg, Campus Marburg, Marburg
| | - Franz Schaefer
- Center for Pediatrics and Adolescent Medicine, Division of Pediatric Nephrology, University Hospital Heidelberg, Heidelberg
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Tuli G, Matarazzo P, de Sanctis L. Clinical Approach to Sodium Homeostasis Disorders in Children with Pituitary-Suprasellar Tumors. Neuroendocrinology 2020; 110:161-171. [PMID: 31401632 DOI: 10.1159/000502609] [Citation(s) in RCA: 2] [Impact Index Per Article: 0.4] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Track Full Text] [Journal Information] [Submit a Manuscript] [Subscribe] [Scholar Register] [Received: 04/06/2019] [Accepted: 08/09/2019] [Indexed: 11/19/2022]
Abstract
Children with pituitary-suprasellar tumors are at high risk of developing sodium metabolism disorders since the tumoral mass itself or surgical and medical treatment can damage AVP release circuits. Additional risk factors are represented by the use of hypotonic fluids, the young age, total parenteral nutrition, and obstructive hydrocephalus secondary to tumor pathology. The most frequent hyponatremic disorders related to AVP in these patients are the syndrome of inappropriate ADH secretion and the cerebral/renal salt wasting syndrome, while hypernatremic conditions include central diabetes insipidus (CDI) and adipsic CDI. The main challenge in the management of these patients is to promptly distinguish the AVP release disorder at the base of the sodium imbalance and treat it correctly by avoiding rapid sodium fluctuations. These disorders can coexist or follow each other in a few hours or days; therefore, careful clinical and biochemical monitoring is necessary, especially during surgery, the use of chemotherapeutic agents, or radiotherapy. This monitoring should be performed by experienced healthcare professionals and should be multidisciplinary, including pediatric endocrinologists, neurosurgeons, and oncologists since maintaining sodium homeostasis also plays a prognostic role in terms of disease survival, therapeutic response, hospitalization rate, and mortality. In this review, we analyze the management of sodium homeostasis disorders in children with pituitary-suprasellar tumors and discuss the main challenges in the diagnosis and treatment of these conditions based on literature data and over 30 years of clinical experience at our Department of Pediatric Endocrinology.
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Affiliation(s)
- Gerdi Tuli
- Department of Pediatric Endocrinology, Regina Margherita Children's Hospital, City of Health and Science University Hospital of Turin, Turin, Italy,
- Department of Public Health and Pediatric Sciences, University of Turin, Turin, Italy,
| | - Patrizia Matarazzo
- Department of Pediatric Endocrinology, Regina Margherita Children's Hospital, City of Health and Science University Hospital of Turin, Turin, Italy
- Department of Public Health and Pediatric Sciences, University of Turin, Turin, Italy
| | - Luisa de Sanctis
- Department of Pediatric Endocrinology, Regina Margherita Children's Hospital, City of Health and Science University Hospital of Turin, Turin, Italy
- Department of Public Health and Pediatric Sciences, University of Turin, Turin, Italy
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Gürbüz F, Taştan M, Turan İ, Yüksel B. Efficiency of Single Dose of Tolvaptan Treatment During the Triphasic Episode After Surgery for Craniopharyngioma. J Clin Res Pediatr Endocrinol 2019; 11:202-206. [PMID: 30257818 PMCID: PMC6571528 DOI: 10.4274/jcrpe.galenos.2018.2018.0182] [Citation(s) in RCA: 10] [Impact Index Per Article: 1.7] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Download PDF] [Figures] [Journal Information] [Submit a Manuscript] [Subscribe] [Scholar Register] [Indexed: 12/01/2022] Open
Abstract
Inappropriate antidiuretic hormone syndrome (SIADH) may develop after intracranial surgery. SIADH in the pediatric age group is usually encountered in patients with an intracranial mass both before and after surgery. Fluid restriction is the standard therapy in SIADH. However, a resistant, hyponatremic pattern may be encountered in some cases. Vaptans have been recently introduced for treatment of hyponatremia due to SIADH. There is inadequate data concerning tolvaptan treatment in pediatric patients. We present a 13 year-old female with SIADH of triphasic episode who was transferred to our clinic after surgery for craniopharyngioma. Resistant hyponatremia did not resolve despite fluid restriction and hypertonic saline support. The patient responded rapidly to a single dose of tolvaptan, with no adverse effect, which resulted in successful control of her SIADH.
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Affiliation(s)
- Fatih Gürbüz
- Çukurova University Faculty of Medicine, Department of Pediatric Endocrinology, Adana, Turkey,* Address for Correspondence: Çukurova University Faculty of Medicine, Department of Pediatric Endocrinology, Adana, Turkey Phone: +90 322 338 60 60 E-mail:
| | - Mehmet Taştan
- Çukurova University Faculty of Medicine, Department of Pediatric Endocrinology, Adana, Turkey
| | - İhsan Turan
- Çukurova University Faculty of Medicine, Department of Pediatric Endocrinology, Adana, Turkey
| | - Bilgin Yüksel
- Çukurova University Faculty of Medicine, Department of Pediatric Endocrinology, Adana, Turkey
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Bardanzellu F, Pintus MC, Masile V, Fanos V, Marcialis MA. Focus on neonatal and infantile onset of nephrogenic syndrome of inappropriate antidiuresis: 12 years later. Pediatr Nephrol 2019; 34:763-775. [PMID: 29546600 DOI: 10.1007/s00467-018-3922-6] [Citation(s) in RCA: 2] [Impact Index Per Article: 0.3] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Track Full Text] [Journal Information] [Submit a Manuscript] [Subscribe] [Scholar Register] [Received: 10/08/2017] [Revised: 02/09/2018] [Accepted: 02/09/2018] [Indexed: 11/25/2022]
Abstract
Nephrogenic syndrome of inappropriate antidiuresis (NSIAD), first described in 2005, is a rare genetic X-linked disease, presenting with hyponatremia, hyposmolarity, euvolemia, inappropriately concentrated urine, increased natriuresis, and undetectable or very low arginine-vasopressine (AVP) circulating levels. It can occur in neonates, infants, or later in life. NSIAD must be early recognized and treated to prevent severe hyponatremia, which can show a dangerous impact on neonatal outcome. In fact, it potentially leads to death or, in case of survival, neurologic sequelae. This review is an update of NSIAD 12 years after the first description, focusing on reported cases of neonatal and infantile onset. The different molecular patterns affecting the AVP receptor 2 (V2R) and determining its gain of function are reported in detail; moreover, we also provide a comparison between the different triggers involved in the development of hyponatremia, the evolution of the symptoms, and modality and efficacy of the different treatments available.
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Affiliation(s)
- Flaminia Bardanzellu
- Neonatal Intensive Care Unit, Neonatal Pathology and Neonatal Section, AOU and University of Cagliari, Cagliari, Italy.
| | - Maria Cristina Pintus
- Neonatal Intensive Care Unit, Neonatal Pathology and Neonatal Section, AOU and University of Cagliari, Cagliari, Italy
| | - Valentina Masile
- Neonatal Intensive Care Unit, Neonatal Pathology and Neonatal Section, AOU and University of Cagliari, Cagliari, Italy
| | - Vassilios Fanos
- Neonatal Intensive Care Unit, Neonatal Pathology and Neonatal Section, AOU and University of Cagliari, Cagliari, Italy
| | - Maria Antonietta Marcialis
- Neonatal Intensive Care Unit, Neonatal Pathology and Neonatal Section, AOU and University of Cagliari, Cagliari, Italy
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Strong A, Muneeruddin S, Parrish R, Lui D, Conley SB. Isosorbide dinitrate in nephronophthisis treatment. Am J Med Genet A 2019; 176:1023-1026. [PMID: 29575630 DOI: 10.1002/ajmg.a.38650] [Citation(s) in RCA: 3] [Impact Index Per Article: 0.5] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 12/19/2017] [Revised: 02/05/2018] [Accepted: 02/05/2018] [Indexed: 12/31/2022]
Abstract
Nephronophthisis is a progressive disease that affects development of the renal tubules and leads to end stage renal disease. Many affected children have isolated renal disease; however, there can be additional manifestations including heart defects, liver fibrosis, brain malformations, and situs inversus. There is no way to slow or modify the disease. We describe a patient who presented at birth with cholestatic jaundice and decreased kidney function, found by exome sequencing to have two NPHP3 variants. Her clinical status deteriorated rapidly, and two disease-modifying agents were given in hopes of slowing disease progression, the arginine vasopressin type II receptor antagonist tolvaptan to stabilize her renal function and isosorbide dinitrate to manage her poorly controlled hypertension. Tolvaptan therapy initiated at 82 days of life had limited effect on the rate of decline in renal function and was insufficient to abrogate the need for dialysis; however, isosorbide dinitrate therapy led to a dramatic improvement in blood pressure control and allowed for the discontinuation of multiple anti-hypertensive agents. This is the first report of the use of tolvaptan and isosorbide dinitrate for nephronophthisis management. We suggest that isosorbide dinitrate may represent a disease-modifying agent in nephronophthisis treatment.
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Affiliation(s)
- Alanna Strong
- Department of Pediatrics, St. Christopher's Hospital for Children, Philadelphia, Pennsylvania
| | - Samina Muneeruddin
- Department of Pediatrics, Section of Nephrology, St. Christopher's Hospital for Children, Philadelphia, Pennsylvania
| | - Richard Parrish
- Department of Pediatrics, Section of Pharmacy, St. Christopher's Hospital for Children, Philadelphia, Pennsylvania
| | - Daniel Lui
- Department of Pediatrics, Section of Pharmacy, St. Christopher's Hospital for Children, Philadelphia, Pennsylvania
| | - Susan B Conley
- Department of Pediatrics, Section of Nephrology, St. Christopher's Hospital for Children, Philadelphia, Pennsylvania
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Kerling A, Toka O, Rüffer A, Müller H, Habash S, Weiss C, Dittrich S, Moosmann J. First experience with Tolvaptan for the treatment of neonates and infants with capillary leak syndrome after cardiac surgery. BMC Pediatr 2019; 19:57. [PMID: 30755181 PMCID: PMC6371520 DOI: 10.1186/s12887-019-1418-6] [Citation(s) in RCA: 7] [Impact Index Per Article: 1.2] [Reference Citation Analysis] [Abstract] [Track Full Text] [Download PDF] [Figures] [Journal Information] [Submit a Manuscript] [Subscribe] [Scholar Register] [Received: 09/05/2018] [Accepted: 01/28/2019] [Indexed: 01/09/2023] Open
Abstract
Background Postoperative fluid management in critically ill neonates and infants with capillary leak syndrome (CLS) and extensive volume overload after cardiac surgery on cardiopulmonary bypass is challenging. CLS is often resistant to conventional diuretic therapy, aggravating the course of weaning from invasive ventilation, increasing length of stay on ICU and morbidity and mortality. Methods Tolvaptan (TLV, vasopressin type 2 receptor antagonist) was used as an additive diuretic in neonates and infants with CLS after cardiac surgery. Retrospective analysis of 25 patients with CLS including preoperative and postoperative parameters was performed. Multivariate regression analysis was performed to identify predictors for TLV response. Results Multivariate analysis identified urinary output during 24 h after TLV administration and mean blood pressure (BP) on day 2 of TLV treatment as predictors for TLV response (AUC = 0.956). Responder showed greater weight reduction (p < 0.0001), earlier weaning from ventilator during TLV (p = 0.0421) and shorter time in the ICU after TLV treatment (p = 0.0155). Serum sodium and serum osmolality increased significantly over time in all patients treated with TLV. Conclusion In neonates and infants with diuretic-refractory CLS after cardiac surgery, additional aquaretic therapy with TLV showed an increase in urinary output and reduction in bodyweight in patients classified as TLV responder. Increase in urinary output and mean BP on day 2 of treatment were strong predictors for TLV response. Electronic supplementary material The online version of this article (10.1186/s12887-019-1418-6) contains supplementary material, which is available to authorized users.
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Affiliation(s)
- Anne Kerling
- Department of Pediatric Cardiology, University of Erlangen-Nürnberg, Loschgestrasse 15, 91054, Erlangen, Germany
| | - Okan Toka
- Department of Pediatric Cardiology, University of Erlangen-Nürnberg, Loschgestrasse 15, 91054, Erlangen, Germany
| | - André Rüffer
- Department of Pediatric Cardiac Surgery, University of Erlangen-Nürnberg, Loschgestrasse 15, 91054, Erlangen, Germany
| | - Hanna Müller
- Department of Pediatrics and Adolescent Medicine, University of Erlangen-Nürnberg, Loschgestrasse 15, 91054, Erlangen, Germany
| | - Sheeraz Habash
- Department of Pediatric Cardiology, University of Erlangen-Nürnberg, Loschgestrasse 15, 91054, Erlangen, Germany
| | - Christel Weiss
- Department of Medical Statistics and Biomathematics, University Hospital Mannheim, University of Heidelberg, Theodor-Kutzer-Ufer 1-3, 68167, Mannheim, Germany
| | - Sven Dittrich
- Department of Pediatric Cardiology, University of Erlangen-Nürnberg, Loschgestrasse 15, 91054, Erlangen, Germany
| | - Julia Moosmann
- Department of Pediatric Cardiology, University of Erlangen-Nürnberg, Loschgestrasse 15, 91054, Erlangen, Germany.
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Tuli G, Tessaris D, Einaudi S, De Sanctis L, Matarazzo P. Tolvaptan Treatment in Children with Chronic Hyponatremia due to Inappropriate Antidiuretic Hormone Secretion: A Report of Three Cases. J Clin Res Pediatr Endocrinol 2017; 9:288-292. [PMID: 28515029 PMCID: PMC5596813 DOI: 10.4274/jcrpe.4531] [Citation(s) in RCA: 20] [Impact Index Per Article: 2.5] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Download PDF] [Figures] [Journal Information] [Submit a Manuscript] [Subscribe] [Scholar Register] [Indexed: 12/12/2022] Open
Abstract
Hyponatremia is the most common electrolyte disorder among hospitalized patients and it is sometimes considered as a poor outcome predictor. Its correction is thus indicated, even in asymptomatic patients. The conventional treatment consists of fluid restriction in presence of euvolemia or hypervolemia; loop diuretics are used in some hypervolemic conditions such as cardiac heart failure, liver cirrhosis and nephrotic syndrome, while intravenous isotonic or hypertonic solutions are administered in hypovolemic conditions. The utilization of demeclocycline and urea is not indicated in pediatric ages due to lack of data on their toxicity and poor tolerance. Recently, a new therapeutic option has been developed, a class of non-peptide arginine vasopressin receptor antagonists called vaptans. Tolvaptan is the only such agent approved in Europe for the treatment of hyponatremia caused by syndrome of inappropriate antidiuretic hormone secretion (SIADH) in adults. In USA, tolvaptan and conivaptan have been approved for treatment of euvolemic and hypervolemic hyponatremia. Few data are so far available in paediatric patients, since only one trial has been registered in Europe which includes children and adolescents, but this trial is still ongoing. Here, we report three children with chronic hyponatremia due to SIADH in which tolvaptan has been used successfully.
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Affiliation(s)
- Gerdi Tuli
- University of Turin, Regina Margherita Children’s Hospital, Department of Public Health and Pediatrics, Division of Pediatric Endocrinology, Turin, Italy
,* Address for Correspondence: University of Turin, Regina Margherita Children’s Hospital, Department of Public Health and Pediatrics, Division of Pediatric Endocrinology, Turin, Italy Phone: + 39 349 3232854 E-mail:
| | - Daniele Tessaris
- University of Turin, Regina Margherita Children’s Hospital, Department of Public Health and Pediatrics, Division of Pediatric Endocrinology, Turin, Italy
| | - Silvia Einaudi
- University of Turin, Regina Margherita Children’s Hospital, Department of Public Health and Pediatrics, Division of Pediatric Endocrinology, Turin, Italy
| | - Luisa De Sanctis
- University of Turin, Regina Margherita Children’s Hospital, Department of Public Health and Pediatrics, Division of Pediatric Endocrinology, Turin, Italy
| | - Patrizia Matarazzo
- University of Turin, Regina Margherita Children’s Hospital, Department of Public Health and Pediatrics, Division of Pediatric Endocrinology, Turin, Italy
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15
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Safety and effectiveness of tolvaptan for fluid management after pediatric cardiovascular surgery. Gen Thorac Cardiovasc Surg 2017. [DOI: 10.1007/s11748-017-0798-5] [Citation(s) in RCA: 10] [Impact Index Per Article: 1.3] [Reference Citation Analysis] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Indexed: 11/26/2022]
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16
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Willemsen RH, Delgado-Carballar V, Elleri D, Thankamony A, Burke GAA, Nicholson JC, Dunger DB. Tolvaptan use during hyperhydration in paediatric intracranial lymphoma with SIADH. Endocrinol Diabetes Metab Case Rep 2016; 2016:EDM160066. [PMID: 27857840 PMCID: PMC5097145 DOI: 10.1530/edm-16-0066] [Citation(s) in RCA: 8] [Impact Index Per Article: 0.9] [Reference Citation Analysis] [Abstract] [Track Full Text] [Download PDF] [Figures] [Journal Information] [Subscribe] [Scholar Register] [Received: 09/23/2016] [Accepted: 09/29/2016] [Indexed: 11/08/2022] Open
Abstract
An 11-year-old boy developed severe syndrome of inappropriate antidiuretic hormone secretion (SIADH) after diagnosis of an intracranial B-cell lymphoma. His sodium levels dropped to 118-120 mmol/L despite 70% fluid restriction. For chemotherapy, he required hyperhydration, which posed a challenge because of severe hyponatraemia. Tolvaptan is an oral, highly selective arginine vasopressin V2-receptor antagonist, which has been licensed in adults for the management of SIADH and has been used in treating paediatric heart failure. Tolvaptan gradually increased sodium levels and allowed liberalisation of fluid intake and hyperhydration. Tolvaptan had profound effects on urinary output in our patient with increases up to 8 mL/kg/h and required close monitoring of fluid balance, frequent sodium measurements and adjustments to intake. After hyperhydration, tolvaptan was stopped, and the lymphoma went into remission with reversal of SIADH. We report one of the first uses of tolvaptan in a child with SIADH, and it was an effective and safe treatment to manage severe SIADH when fluid restriction was not possible or effective. However, meticulous monitoring of fluid balance and sodium levels and adjustments of fluid intake are required to prevent rapid sodium changes. LEARNING POINTS Tolvaptan can be used in paediatric patients with SIADH to allow hyperhydration during chemotherapy.Tolvaptan has profound effects on urinary output and meticulous monitoring of fluid balance and sodium
levels is therefore warranted.Tolvaptan was well tolerated without significant side effects.
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Affiliation(s)
- Ruben H Willemsen
- Department of Paediatric Endocrinology , University of Cambridge, Cambridge , UK
| | | | - Daniela Elleri
- Department of Paediatric Endocrinology , University of Cambridge, Cambridge , UK
| | - Ajay Thankamony
- Department of Paediatric Endocrinology , University of Cambridge, Cambridge , UK
| | - G A Amos Burke
- Department of Paediatric Haematology and Oncology , Addenbrooke's Hospital, Cambridge University Hospitals NHS Foundation Trust, Cambridge , UK
| | - James C Nicholson
- Department of Paediatric Haematology and Oncology , Addenbrooke's Hospital, Cambridge University Hospitals NHS Foundation Trust, Cambridge , UK
| | - David B Dunger
- Department of Paediatric Endocrinology , University of Cambridge, Cambridge , UK
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Abstract
OBJECTIVES Focusing on critically ill children with cardiac disease, we will review common causes of fluid perturbations, clinical recognition, and strategies to minimize and treat fluid-related complications. DATA SOURCE MEDLINE and PubMed. CONCLUSIONS Meticulous fluid management is vital in critically ill children with cardiac disease. Fluid therapy is important to maintain adequate blood volume and perfusion pressure in order to support cardiac output, tissue perfusion, and oxygen delivery. However, fluid overload and acute kidney injury are common and are associated with increased morbidity and mortality. Understanding the etiologies for disturbances in volume status and the pathophysiology surrounding those conditions is crucial for providing optimal care.
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18
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Hypertonic saline with furosemide for diuretic resistant congestive heart failure in an infant. Int J Cardiol 2016; 215:127-8. [PMID: 27111174 DOI: 10.1016/j.ijcard.2016.04.059] [Citation(s) in RCA: 1] [Impact Index Per Article: 0.1] [Reference Citation Analysis] [Key Words] [Track Full Text] [Journal Information] [Submit a Manuscript] [Subscribe] [Scholar Register] [Received: 02/08/2016] [Accepted: 04/11/2016] [Indexed: 11/22/2022]
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19
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Schranz D, Voelkel NF. "Nihilism" of chronic heart failure therapy in children and why effective therapy is withheld. Eur J Pediatr 2016; 175:445-55. [PMID: 26895877 PMCID: PMC4806719 DOI: 10.1007/s00431-016-2700-3] [Citation(s) in RCA: 39] [Impact Index Per Article: 4.3] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Track Full Text] [Download PDF] [Figures] [Journal Information] [Submit a Manuscript] [Subscribe] [Scholar Register] [Received: 08/17/2015] [Revised: 12/06/2015] [Accepted: 01/26/2016] [Indexed: 12/11/2022]
Abstract
UNLABELLED Major advances in chronic heart failure (cHF) therapy have been achieved and documented in adult patients, while research regarding the mechanisms and therapy of cHF in children has lagged behind. Based on receptor physiological studies and pharmacological knowledge, treatment with specific ß1-adrenergic receptor blocker (ARB), tissue angiotensin-converting enzyme inhibitor (ACE-I), and mineralocorticoid antagonists have to be recommended in children despite lack of sufficient data derived from prospective randomized studies. At our institution, bisoprolol, lisinopril, and spironolactone have been firmly established to treat systolic cHF, hypoplastic left heart syndrome (HLHS) following hybrid approach and congenital left-right shunt diseases, latest in patients where surgery has to be delayed. Chronic therapy with long-acting diuretics and fluid restriction are not advocated because short-term effects are achieved at the expense of further neuro-humoral stimulation. It remains unclear why diuretics are recommended although evidence-based studies, documenting long-term benefit, are missing. However, that is true for all currently used drugs for pediatric cHF. CONCLUSION This review focuses on the prevailing "nihilism" of cHF therapy in children with the goal to encourage physicians to treat pediatric cHF with a rationally designed therapy, which combines available agents that have been shown to improve survival in adult patients with cHF. Because of the lack of clinical trials, which generate the needed evidence, surrogate variables like heart and respiratory rate, weight gain, image-derived data, and biomarkers should be monitored and used instead. The recommended pharmacological therapy for systolic heart failure is also provided as the basis for utilizing reversible pulmonary arterial banding (PAB) as a novel strategy in young children with dilative cardiomyopathy (DCM) with preserved right ventricular function. WHAT IS KNOWN • Heart failure (HF) in children is a serious public health concern. • HF has numerous etiologies, but unspecific symptoms. • HF interplays among neuro-humoral, and molecular abnormalities. • Pediatric cHF-therapy is currently based on loop-diuretics, fluid restriction and digoxin. What is New: • Cardiac function analysis has to include cardiac synchrony and VVI. • Considering enormous potential of cardiac regeneration, therapy has to extend with selective ß1-ARB, tissue ACE-I and mineralocorticoid blockers, loop-diuretics avoided as ever possible. • Inhibition of the endogenous neuro-humoral stimulation is monitored by surrogate parameters as heart and breath rate and systolic and diastolic blood pressure. • Advocated HF therapy serves for regenerative strategies as reversible Pulmonary Artery Banding in DCM.
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Affiliation(s)
- Dietmar Schranz
- />Pediatric Heart Center, Justus-Liebig University Clinic, Feulgenstr. 12, 30385 Giessen, Germany
| | - Norbert F. Voelkel
- />School of Pharmacy, Virginia Commonwealth University, Richmond, VA USA
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20
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Pediatric Cardiac Intensive Care Society 2014 Consensus Statement: Pharmacotherapies in Cardiac Critical Care Fluid Management. Pediatr Crit Care Med 2016; 17:S35-48. [PMID: 26945328 DOI: 10.1097/pcc.0000000000000633] [Citation(s) in RCA: 28] [Impact Index Per Article: 3.1] [Reference Citation Analysis] [Abstract] [Track Full Text] [Journal Information] [Submit a Manuscript] [Subscribe] [Scholar Register] [Indexed: 01/11/2023]
Abstract
OBJECTIVE In this Consensus Statement, we review the etiology and pathophysiology of fluid disturbances in critically ill children with cardiac disease. Clinical tools used to recognize pathologic fluid states are summarized, as are the mechanisms of action of many drugs aimed at optimal fluid management. DATA SOURCES The expertise of the authors and a review of the medical literature were used as data sources. DATA SYNTHESIS The authors synthesized the data in the literature in order to present clinical tools used to recognize pathologic fluid states. For each drug, the physiologic rationale, mechanism of action, and pharmacokinetics are synthesized, and the evidence in the literature to support the therapy is discussed. CONCLUSIONS Fluid management is challenging in critically ill pediatric cardiac patients. A myriad of causes may be contributory, including intrinsic myocardial dysfunction with its associated neuroendocrine response, renal dysfunction with oliguria, and systemic inflammation with resulting endothelial dysfunction. The development of fluid overload has been associated with adverse outcomes, including acute kidney injury, prolonged mechanical ventilation, increased vasoactive support, prolonged hospital length of stay, and mortality. An in-depth understanding of the many factors that influence volume status is necessary to guide optimal management.
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21
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Brissaud O, Botte A, Cambonie G, Dauger S, de Saint Blanquat L, Durand P, Gournay V, Guillet E, Laux D, Leclerc F, Mauriat P, Boulain T, Kuteifan K. Experts' recommendations for the management of cardiogenic shock in children. Ann Intensive Care 2016; 6:14. [PMID: 26879087 PMCID: PMC4754230 DOI: 10.1186/s13613-016-0111-2] [Citation(s) in RCA: 22] [Impact Index Per Article: 2.4] [Reference Citation Analysis] [Abstract] [Track Full Text] [Download PDF] [Figures] [Journal Information] [Subscribe] [Scholar Register] [Received: 08/31/2015] [Accepted: 01/26/2016] [Indexed: 12/13/2022] Open
Abstract
Cardiogenic shock which corresponds to an acute state of circulatory failure due to impairment of myocardial contractility is a very rare disease in children, even more than in adults. To date, no international recommendations regarding its management in critically ill children are available. An experts’ recommendations in adult population have recently been made (Levy et al. Ann Intensive Care 5(1):52, 2015; Levy et al. Ann Intensive Care 5(1):26, 2015). We present herein recommendations for the management of cardiogenic shock in children, developed with the grading of recommendations’ assessment, development, and evaluation system by an expert group of the Groupe Francophone de Réanimation et Urgences Pédiatriques (French Group for Pediatric Intensive Care and Emergencies). The recommendations cover four major fields of application such as: recognition of early signs of shock and the patient pathway, management principles and therapeutic goals, monitoring hemodynamic and biological variables, and circulatory support (indications, techniques, organization, and transfer criteria). Major principle care for children with cardiogenic shock is primarily based on clinical and echocardiographic assessment. There are few drugs reported as effective in childhood in the medical literature. The use of circulatory support should be facilitated in terms of organization and reflected in the centers that support these children. Children with cardiogenic shock are vulnerable and should be followed regularly by intensivist cardiologists and pediatricians. The experts emphasize the multidisciplinary nature of management of children with cardiogenic shock and the importance of effective communication between emergency medical assistance teams (SAMU), mobile pediatric emergency units (SMUR), pediatric emergency departments, pediatric cardiology and cardiac surgery departments, and pediatric intensive care units.
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Affiliation(s)
- Olivier Brissaud
- Unité de Réanimation Pédiatrique et Néonatale, Hôpital des Enfants, CHU Pellegrin Enfants, Place Amélie Raba Léon, 33000, Bordeaux, France.
| | - Astrid Botte
- Unité de Réanimation Pédiatrique, Faculté de Médecine, Université de Lille Nord de France, Hôpital Jeanne de Flandre CHU de Lille, 54, Avenue Eugène Avinée, 59037, Lille Cedex, France
| | - Gilles Cambonie
- Département de Pédiatrie Néonatale et Réanimations, Pôle Hospitalo-Universitaire Femme-Mère-Enfant, Hôpital Arnaud-de-Villeneuve, Centre Hospitalier Régional Universitaire de Montpellier, 371, Avenue du Doyen-Gaston-Giraud, 34295, Montpellier Cedex 5, France
| | - Stéphane Dauger
- Réanimation et Surveillance Continue Pédiatriques, Pôle de Pédiatrie Médicale, Hôpital Robert-Debré, Assistance Publique-Hôpitaux de Paris, Université Paris Diderot-Paris 7, Sorbonne Paris Cité, 48, Boulevard Sérurier, 75019, Paris, France
| | - Laure de Saint Blanquat
- Service de Réanimation, CHU Necker-Enfants-Malades, 149, rue de Sèvres, 75743, Paris Cedex 15, France
| | - Philippe Durand
- Réanimation Pédiatrique, AP-HP, CHU Kremlin Bicêtre, 78, rue du Général Leclerc, 94270, Le Kremlin Bicêtre, France
| | - Véronique Gournay
- Service de Cardiologie Pédiatrique, CHU de Nantes, 44093, Nantes Cedex, France
| | - Elodie Guillet
- Unité de Réanimation Pédiatrique et Néonatale, Hôpital des Enfants, CHU Pellegrin Enfants, Place Amélie Raba Léon, 33000, Bordeaux, France
| | - Daniela Laux
- Pôle des Cardiopathies Congénitales, Centre Chirurgical Marie Lannelongue, 133, Avenue de la Résistance, 92350, Le Plessis-Robinson, France
| | - Francis Leclerc
- Unité de Réanimation Pédiatrique, Faculté de Médecine, Université de Lille Nord de France, Hôpital Jeanne de Flandre CHU de Lille, 54, Avenue Eugène Avinée, 59037, Lille Cedex, France
| | - Philippe Mauriat
- Service de Cardiologie Pédiatrique et Congénitale, Hôpital Haut-Lévèque, CHU de Bordeaux, Avenue de Magellan, 33604, Pessac Cedex, France
| | - Thierry Boulain
- Service de Réanimation Polyvalente, Hôpital de La Source, Centre Hospitalier Régional Orléans, 45067, Orléans, France
| | - Khaldoun Kuteifan
- Service de Réanimation Médicale, Hôpital Émile-Muller, 68070, Mulhouse, France
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22
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Higashi K, Murakami T, Ishikawa Y, Itoi T, Ohuchi H, Kodama Y, Honda T, Masutani S, Yamazawa H, Senzaki H, Ishikawa S. Efficacy and safety of tolvaptan for pediatric patients with congestive heart failure. Multicenter survey in the working group of the Japanese Society of PEdiatric Circulation and Hemodynamics (J-SPECH). Int J Cardiol 2016; 205:37-42. [DOI: 10.1016/j.ijcard.2015.12.003] [Citation(s) in RCA: 20] [Impact Index Per Article: 2.2] [Reference Citation Analysis] [Track Full Text] [Journal Information] [Submit a Manuscript] [Subscribe] [Scholar Register] [Received: 07/22/2015] [Revised: 11/11/2015] [Accepted: 12/12/2015] [Indexed: 11/16/2022]
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23
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Sato N, Sugiura T, Nagasaki R, Suzuki K, Ito K, Kato T, Inukai S, Saitoh S. Effects of tolvaptan on congestive heart failure complicated with chylothorax in a neonate. Pediatr Int 2015; 57:1020-2. [PMID: 26508187 DOI: 10.1111/ped.12697] [Citation(s) in RCA: 7] [Impact Index Per Article: 0.7] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Journal Information] [Submit a Manuscript] [Subscribe] [Scholar Register] [Received: 09/05/2014] [Revised: 03/12/2015] [Accepted: 03/26/2015] [Indexed: 11/30/2022]
Abstract
Tolvaptan is an oral vasopressin type 2 receptor antagonist that can be used for heart failure patients with hyponatremia or symptomatic congestion. Although the effects of tolvaptan in adults have been well documented, only limited information is available in children. The case of a neonate with congestive heart failure complicated with chylothorax after palliative surgery for transposition of the great arteries treated with tolvaptan is reported. Slow up-titration to 0.1 mg/kg successfully increased urine output and improved refractory congestive heart failure without hypernatremia. Subsequently, bodyweight and chylothorax decreased gradually. Moreover, the use of tolvaptan reduced the dosage of furosemide. Tolvaptan could be an alternative drug for neonates with congestive heart failure. Further large studies are needed to confirm the efficacy and identify the appropriate dose of tolvaptan in neonates.
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Affiliation(s)
- Nikiko Sato
- Department of Pediatrics and Neonatology, Graduate School of Medical Sciences Nagoya City University, Nagoya, Japan
| | - Tokio Sugiura
- Department of Pediatrics and Neonatology, Graduate School of Medical Sciences Nagoya City University, Nagoya, Japan
| | - Rika Nagasaki
- Department of Pediatrics and Neonatology, Graduate School of Medical Sciences Nagoya City University, Nagoya, Japan
| | - Kazutaka Suzuki
- Department of Pediatrics and Neonatology, Graduate School of Medical Sciences Nagoya City University, Nagoya, Japan
| | - Koichi Ito
- Department of Pediatrics and Neonatology, Graduate School of Medical Sciences Nagoya City University, Nagoya, Japan
| | - Takenori Kato
- Department of Pediatrics and Neonatology, Graduate School of Medical Sciences Nagoya City University, Nagoya, Japan
| | - Sachiko Inukai
- Department of Pediatrics and Neonatology, Graduate School of Medical Sciences Nagoya City University, Nagoya, Japan
| | - Shinji Saitoh
- Department of Pediatrics and Neonatology, Graduate School of Medical Sciences Nagoya City University, Nagoya, Japan
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24
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Sami SA, Moffett BS, Karlsten ML, Cabrera AG, Price JF, Dreyer WJ, Denfield SW, Jeewa A. Novel Use of Tolvaptan in a Pediatric Patient With Congestive Heart Failure Due to Duchenne Muscular Dystrophy and Congenital Adrenal Hyperplasia. J Pediatr Pharmacol Ther 2015; 20:393-6. [DOI: 10.5863/1551-6776-20.5.393] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Indexed: 11/11/2022]
Abstract
Successful management of hyponatremia in heart failure patients requires a multifaceted approach in order to preserve end-organ function. We describe the novel use of a selective vasopressin receptor antagonist, tolvaptan, for management of hyponatremia in a 17-year-old Caucasian male with severe Duchenne muscular dystrophy, congestive heart failure (CHF), and congenital adrenal hyperplasia. The medical history was significant for recurrent admissions for hyponatremia secondary to adrenal crises, which was also exacerbated by his CHF. After initiation of tolvaptan and its extended administration, he had no further hyponatremia-related admissions and no adverse reactions. The complexity of this combination of conditions is presented, and the efficacy of the drug and the rationale behind the treatment approach is discussed.
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Affiliation(s)
- Sarah A. Sami
- Baylor College of Medicine, Department of Pediatrics, Section of Pediatric Cardiology, Houston, Texas
| | - Brady S. Moffett
- Texas Children's Hospital, Department of Pharmacy, Houston, Texas
| | - Melissa L. Karlsten
- Baylor College of Medicine, Department of Pediatrics, Section of Pediatric Cardiology, Houston, Texas
| | - Antonio G. Cabrera
- Baylor College of Medicine, Department of Pediatrics, Section of Pediatric Cardiology, Houston, Texas
| | - Jack F. Price
- Baylor College of Medicine, Department of Pediatrics, Section of Pediatric Cardiology, Houston, Texas
| | - William J. Dreyer
- Baylor College of Medicine, Department of Pediatrics, Section of Pediatric Cardiology, Houston, Texas
| | - Susan W. Denfield
- Baylor College of Medicine, Department of Pediatrics, Section of Pediatric Cardiology, Houston, Texas
| | - Aamir Jeewa
- Baylor College of Medicine, Department of Pediatrics, Section of Pediatric Cardiology, Houston, Texas
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25
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Filippatos TD, Elisaf MS. Hyponatremia in patients with heart failure. World J Cardiol 2013; 5:317-328. [PMID: 24109495 PMCID: PMC3783984 DOI: 10.4330/wjc.v5.i9.317] [Citation(s) in RCA: 48] [Impact Index Per Article: 4.0] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Download PDF] [Journal Information] [Submit a Manuscript] [Subscribe] [Scholar Register] [Received: 06/23/2013] [Revised: 07/30/2013] [Accepted: 08/17/2013] [Indexed: 02/06/2023] Open
Abstract
The present review analyses the mechanisms relating heart failure and hyponatremia, describes the association of hyponatremia with the progress of disease and morbidity/mortality in heart failure patients and presents treatment options focusing on the role of arginine vasopressin (AVP)-receptor antagonists. Hyponatremia is the most common electrolyte disorder in the clinical setting and in hospitalized patients. Patients with hyponatremia may have neurologic symptoms since low sodium concentration produces brain edema, but the rapid correction of hyponatremia is also associated with major neurologic complications. Patients with heart failure often develop hyponatremia owing to the activation of many neurohormonal systems leading to decrease of sodium levels. A large number of clinical studies have associated hyponatremia with increased morbidity and mortality in patients hospitalized for heart failure or outpatients with chronic heart failure. Treatment options for hyponatremia in heart failure, such as water restriction or the use of hypertonic saline with loop diuretics, have limited efficacy. AVP-receptor antagonists increase sodium levels effectively and their use seems promising in patients with hyponatremia. However, the effects of AVP-receptor antagonists on hard outcomes in patients with heart failure and hyponatremia have not been thoroughly examined.
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