Practice Patterns of Physician Treatment for Pediatric Chronic Myelogenous Leukemia

Abstract

Chronic myelogenous leukemia (CML) is a rare disease in children for which pediatric evidence-based guidelines are lacking. We designed an anonymous survey for practicing pediatric oncologists and bone marrow transplantation (BMT) physicians to assess their willingness to recommend BMT for a patient with CML based on various clinical scenarios. A total of 274 physicians responded to the survey (13.4% response rate). Nearly all pediatric oncologists and BMT physicians recommended against BMT at time of diagnosis of CML in the chronic phase, with only 8.0% and 1.9% recommending BMT if a matched sibling donor (MSD) and a matched unrelated donor (MUD), respectively, was available. Similarly, after a first poor response to tyrosine kinase inhibitor (TKI) therapy or hematologic relapse, physicians continued to recommend against BMT (39.5% and 23.3% recommended BMT in patients with a matched sibling donor and matched unrelated donor, respectively). However, 81.7% and 69.8% of respondents would recommend BMT after 2 hematologic relapses on TKI therapy, if an MSD and an MUD, respectively, were available. In addition, there was great interest in developing a clinical trial evaluating the safety and efficacy of stopping TKIs in children with CML who achieve and maintain a deep molecular response, with 86.7% of respondents stating they would offer such a trial to their pediatric patients. This survey highlights the need for evidence-based, pediatric-specific guidelines for the management of children and adolescents with CML.

The Contemporary Role of Hematopoietic Stem Cell Transplantation in the Management of Chronic Myeloid Leukemia: Is It the Same in All Settings?

“…A survey of pediatric oncologists and HSCT physicians revealed that HSCT preference for pediatric CML is a minority, yet they were interested in a DMR-driven discontinuation trial [50]. Lifelong TKI treatment demands, especially from childhood to young adulthood, could lead to growth issues, potentially favoring HSCT.…”

Children and Adolescents with Chronic Myeloproliferative Neoplasms: Still an Unmet Biological and Clinical Need?

“…A recent comprehensive survey of 247 US-based pediatric hematologists' attitude towards an optimal treatment algorithm suggest that most do not support the use of allo-SCT for first-line therapy, and about 40% support it if suitable donor was available. 35 However, the vast majority support the use of allo-SCT following 2 lines of TKI therapy.…”

“…Allo‐SCT should also be offered to CML patients who acquire the T315I mutation in BCR‐ABL1 , since the third generation TKI, ponatinib, is not currently licensed for children and adolescents. A recent comprehensive survey of 247 US‐based pediatric hematologists’ attitude towards an optimal treatment algorithm suggest that most do not support the use of allo‐SCT for first‐line therapy, and about 40% support it if suitable donor was available 35 . However, the vast majority support the use of allo‐SCT following 2 lines of TKI therapy.…”

An intelligent control strategy for cancer cells reduction in patients with chronic myelogenous leukaemia using the reinforcement learning and considering side effects of the drug

“…A survey was performed on a physician's idea about bone marrow transplant for CML detection among children, in the north of America. In this survey, 274 physicians have participated; about 7.8% of participants have recommended the bone marrow transplant for CML detection among children) Andolina et al, 2019(. Data mining methods have been used for different types of data related to patients with CML to detect potential targets (Benard, Gentles, Köhnke, Majeti, & Thomas, 2019).…”