Peer-review started: November 5, 2017
First decision: November 20, 2017
Revised: November 27, 2017
Accepted: December 6, 2017
Article in press: December 6, 2017
Published online: February 2, 2018
Panniculitis is a rare manifestation of an alpha-1 antitrypsin deficiency (AATD), an already rare disease. This rarity has created a paucity in the data concerning effective management strategies of AATD-panniculitis.
This systematic review has investigated the available literature for the optimal management strategies for clinical resolution of AATD-panniculitis. Effective management will be helpful for both patients and clinicians, as well as opening up avenues of research allowing better understanding of this disease process.
To perform a systematic review of the management of patients with AATD-panniculitis, investigating interventions used and whether clinical resolution was achieved. This objective was realised, enabling the authors to advise an effective management strategy for clinical resolution. Successful management will be helpful for both patients and clinicians, as well as opening up avenues of research allowing better understanding of this disease process.
A variety of databases were searched for appropriate papers. A title and abstract review was performed by two reviewers, and pertinent studies were obtained in full - to include 32 case reports and 4 case series. The appropriate data was extracted by one reviewer and checked by another. The nature of the data meant that only narrative synthesis was performed, and a formal assessment of quality was not applicable.
Augmentation therapy was the most successful treatment, achieving clinical resolution in 100% of managed patients. Dapsone was a cheaper alternative, achieving clinical resolution in 62%, but being very poorly tolerated. Other single-agent antibiotics were mostly unsuccessful, with the exception of doxycycline (complete resolution in 33%). Immunosuppressants were also largely unsuccessful; 80% of patients exhibited no response. Liver transplantation and therapeutic plasma exchange displayed complete resolution in 66% of patients. Other strategies, such as non-steroidal anti-inflammatory drugs (NSAIDs), or combination antibiotics, did not show sufficient response rates to recommend their use. These results contribute valuable information on effective therapeutic strategies for AATD-panniculitis. Further understanding on the mechanisms of certain therapeutic agents (e.g., dapsone/immunosuppressants) are yet to be investigated, and might unlock key concepts on the pathogenesis of panniculitis or AATD.
This study suggests an effective management strategy for panniculitis presenting in AATD. Based on likelihood of complete clinical resolution and cost-implications, AATD-panniculitis should be managed with dapsone first-line, and augmentation as a second-line measure. In countries where augmentation is not available, or in cases of rapidly progressing disease, plasmapheresis may be a valuable alternative.
This study has the potential to change the future management of AATD-panniculitis, both unifying an effective management strategy amongst current/future clinicians, and opening up avenues of further research to enable better understanding of this disease presentation