Cases of Patients Treated in Countries With Limited Resources and Discussed by Experts of the International CML Foundation (iCMLf)—Case No. 2: Treatment-Free Remission After 9 Years of Imatinib Treatment Without Prior Achievement of Sustained Deep Molecular Response

Abstract

Pediatric chronic myeloid leukemia (pCML) is a rare malignancy that nowadays is treated upfront with tyrosine kinase inhibitors (TKIs). As demonstrated in adult CML patients, achieving deep molecular response (DMR) and maintaining this status over 2 years results in the opportunity to discontinue TKI therapy. Following cessation, this treatment-free remission (TFR) status is successfully achieved by approximately 50% of the patients, while the other half experience molecular relapse within ≤ 6 months, requiring a TKI restart. As pCML accounts for only 2%–3% of all childhood leukemias, experience and familiarity with this disease, especially with stopping attempts, are still very limited. Small pCML cohorts enrolled in stopping TKI trials, with strict criteria applied for both depth and maintenance of DMR, have demonstrated the achievable TFR success rates seem comparable to adults. However, recommendations for considering TFR in pCML have yet to be defined. We report on a 9-year-old Brazilian boy diagnosed with CML in a chronic phase. He was treated with imatinib and achieved a molecular response (BCR::ABL1 transcript rate < 0.1%) at Month 12. Not achieving DMR, he responded well, but not optimally, to TKI therapy. Contrary to existing guidelines on TKI cessation in adults, after 9 years, imatinib was stopped. With a follow-up of 24 months, the patient is in TFR and now maintains DMR successfully. With the support of the International CML Foundation (iCMLf), which aims to improve outcomes for CML patients globally, this rare case from Brazil is discussed from the perspective of a pediatric hemato-oncologist from a high-income country, a pediatric hemato-oncologist from a low- and middle-income country, an adult CML hematologist, and the treating physician. Sharing cases of pCML in LMICs and highlighting the resources offered by the iCMLf, particularly the Knowledge Center (available online), will hopefully improve the expertise on pCML treatment worldwide.