Opinion Review
Copyright ©The Author(s) 2020. Published by Baishideng Publishing Group Inc. All rights reserved.
World J Clin Oncol. Jun 24, 2020; 11(6): 308-319
Published online Jun 24, 2020. doi: 10.5306/wjco.v11.i6.308
Horn of plenty: Value of the international registry for pediatric chronic myeloid leukemia
Meinolf Suttorp, Markus Metzler, Frédéric Millot
Meinolf Suttorp, Pediatric Hemato-Oncology, Medical Faculty, Technical University Dresden, Dresden D-01307, Germany
Markus Metzler, Pediatric Oncology and Hematology, Department of Pediatrics and Adolescent Medicine, University Hospital Erlangen, Erlangen D-9105, Germany
Frédéric Millot, Inserm CIC 1402, University Hospital Poitiers, Poitiers F-86000, France
Author contributions: All authors developed the concept of this manuscript, treated individual patients, communicated in their role as study chair with treatment centers enrolling patients into the network, performed data analysis and supervised therapeutic approaches, revised the text and approved the final and revised version of the manuscript; Suttorp M wrote the first draft of the manuscript and all authors critically discussed the content; Millot F assured data collection and data management.
Conflict-of-interest statement: Suttorp M and Metzler M have received honoraria for consulting and lectures from Novartis Pharma and BMS as well as research grants (institutional) from Novartis Pharma; Millot F has nothing to disclose.
Open-Access: This article is an open-access article that was selected by an in-house editor and fully peer-reviewed by external reviewers. It is distributed in accordance with the Creative Commons Attribution NonCommercial (CC BY-NC 4.0) license, which permits others to distribute, remix, adapt, build upon this work non-commercially, and license their derivative works on different terms, provided the original work is properly cited and the use is non-commercial. See: http://creativecommons.org/licenses/by-nc/4.0/
Corresponding author: Meinolf Suttorp, MD, PhD, Emeritus Professor, Research Scientist, Senior Lecturer, Senior Scientist, Pediatric Hemato-Oncology, Medical Faculty, Technical University Dresden, Fetscherstr 74, Dresden D-01307, Germany. meinolf.suttorp@uniklinikum-dresden.de
Received: December 13, 2019
Peer-review started: December 13, 2019
First decision: April 22, 2020
Revised: May 18, 2020
Accepted: May 29, 2020
Article in press: May 29, 2020
Published online: June 24, 2020
Processing time: 194 Days and 2.4 Hours
Abstract

Chronic myeloid leukemia (CML) in minors is a rare disease which can be effectively treated by tyrosine kinase inhibitors (TKIs) since the year 2000. A majority of pediatricians will encounter one or two CML patients in the course of their careers and will typically have to rely on written information along with their own intuition to provide care. Knowledge of response to TKIs and of age-specific side effects has an impact on the design of pediatric CML trials in many ways aiming to contribute toward greater predictability of clinical improvements. Information from a registry on a rare disease like CML offers the enormous benefit of enabling treating physicians to interact and share their collective experience. The International Registry on Pediatric CML (IR-PCML) was founded at Poitiers/France almost 10 years ago. Since then, the number of collaboration centers and in parallel of registered patients continuously increased (> 550 patients as of December 2019). Ideally, from a given treatment center in a country data are transferred to a national coordinator who interacts with the IR-PCML. In the sense of quality assurance, the registry can offer dissemination of knowledge on state-of-the-art diagnostics (including reference appraisal), optimal treatment approaches, and follow-up procedures within a network that is exerting its strength via participation. With continuous growth during the recent years, very rare subgroups of patients could be identified (e.g., CML diagnosed at age < 3 years, children presenting with specific problems at diagnosis or during course of treatment) which had not been described before. Publications coming from the IR-PCML disseminated this useful information derived from patients who robustly participate and share information about their disease, among themselves and with their caregivers and clinicians. Patient input driving the collection of data on this rare leukemia is the basis for the considerable success of bringing new therapeutics into clinical use.

Keywords: Pediatric chronic myeloid leukemia; International registry; Rare disease; Collaboration and data exchange

Core tip: Chronic myeloid leukemia (CML) in minors is a rare disease which can be effectively treated by tyrosine kinase inhibitors since the year 2000. Publications coming from the International Registry CML disseminated this useful information derived from patients who robustly participate and share information about their disease, among themselves and with their caregivers and clinicians. Patient input driving the collection of data on this rare leukemia is the basis for the considerable success of bringing new therapeutics into clinical use.