Outcomes of Allogeneic Hematopoietic Cell Transplantation in Children and Young Adults with Chronic Myeloid Leukemia: A CIBMTR Cohort Analysis

Chaudhury, Sonali; Sparapani, Rodney; Hu, Zhen; Nishihori, Taiga; Abdel‐Azim, Hisham; Malone, Adriana K.; Olsson, Richard F.; Hamadani, Mehdi; Daly, Andrew; Bacher, Ulrike; Wirk, Baldeep; Kamble, Rammurti T.; Gale, Robert Peter; Wood, William A.; Hale, Gregory A.; Wiernik, Peter H.; Hashmi, Shahrukh K.; Marks, David I.; Üstün, Celalettin; Munker, Reinhold; Savani, Bipin N.; Alyea, Edwin P.; Popat, Uday; Sobecks, Ronald; Kalaycio, Matt; Maziarz, Richard T.; Hijiya, Nobuko; Saber, Wael

doi:10.1016/j.bbmt.2016.02.015

Cited by 27 publications

(28 citation statements)

References 54 publications

(62 reference statements)

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“…The quality of life is one of the integral parts of the survival outcomes especially in pediatric populations, which is to a big extent related to the incidence and severity of cGVHD, and we confirmed a significant higher incidence of cGVHD with peripheral blood stem cell source than the marrow source …”

Section: Discussionsupporting

confidence: 71%

“…As described in the results section, the 5‐year EFS in our study cohort was 80% and was significantly better with peripheral blood stem cell source ( P = .05), but we could not detect an impact of disease stage, time to transplant, and donor gender which was reported among other different studies . Also this study reported total cumulative incidence of relapse of 16%, which was lower than that reported by other pediatric studies, and of interest about 87% of them maintained their molecular remission with salvage therapies post‐transplant …”

Section: Discussioncontrasting

confidence: 67%

“…But a large retrospective analysis from CIBMTR reviewed the outcome of HSCT in children and young adults and reported 5‐year OS and LFS of 83% and 64%, respectively, with MSD, and the 5‐year OS and LFS were 68% and 59%, respectively, with MUD. The reported cumulative incidence of TRM within this entire cohort was 13% at 1 year with significant increased risk with the use of unrelated donors ( P < .001) and PBSCs ( P = .003) . Thus, the main challenge for improvement the HSCT outcome was to reduce the TRM through better donor selection, and stem cell source in addition to advances in supportive care measures for the HSCT recipients.…”

Section: Discussionmentioning

confidence: 93%

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Outcomes of allogenic hematopoietic cell transplantation for childhood chronic myeloid leukemia: Single‐center experience

Hafez

Abdallah

Hammad

et al. 2020

Pediatric Transplantation

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Background/objectives: Despite the apparent efficacy and favorable toxicity profile of TKIs, allogeneic SCT remains the only curative treatment for CML especially in younger patients, but TRM should be considered. We evaluated the clinical outcomes of pediatric CML patients who had SCT in our center. Methods: This retrospective study included children with CML, who received an allogeneic SCT at Children Cancer Hospital Egypt, 57357, from 2007 to 2017. All patients received myeloablative conditioning chemotherapy containing busulfan/cyclophosphamide followed by stem cell infusion from MRD. Results: From 121 patients diagnosed with CML, 43 had available MRD and subjected to HSCT while 78 patients continued TKI therapy. The median time to transplant from diagnosis was 13 months. At initial diagnosis, there were 39 patients in CP and 4 had blastic crises. Bone marrow harvest was the stem cell source in 32 patients, while 11 cases received mobilized peripheral blood stem cells with average stem cell dose of 4.45 × 10 6 /kg. The probabilities of overall survival and event-free survivalat 5 years were 97.4% and 79.8%, respectively. TRM at 100 days and TRM at 1-year post-transplant were 0%. The incidence of chronic GVHD was significantly higher in peripheral blood than bone marrow stem cell source (P = .004). Conclusion:Considering the excellent survival rates and very low TRM, HSCT is still a valid option for pediatric patients with newly diagnosed CML with best using marrow stem cell source to avoid a significant risk of cGVHD and its related complications. K E Y W O R D Schronic myeloid leukemia, hematopoietic stem cell transplantation, transplant-related mortality, tyrosine kinase inhibitors

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Section: Discussionsupporting

confidence: 71%

Section: Discussioncontrasting

confidence: 67%

Section: Discussionmentioning

confidence: 93%

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Outcomes of allogenic hematopoietic cell transplantation for childhood chronic myeloid leukemia: Single‐center experience

Hafez

Abdallah

Hammad

et al. 2020

Pediatric Transplantation

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“…88 With its more aggressive clinical features and evidence of biological and host differences, pediatric CML cannot be easily categorized using adult criteria or treated using adult guidelines. As the only established therapeutic approach that can achieve a lifelong remission in CML, allogeneic BMT may allow pediatric CML patients to avoid lifelong TKI therapy.…”

mentioning

confidence: 99%

“…88,91,93,94 Late outcomes of BMT (.2 years) for CML have been evaluated in the Bone Marrow Transplant Survivor study, 95 although only 5% of the patients were ,21 years of age. In that study, overall health was excellent, very good, or good in 78% of patients.…”

mentioning

confidence: 99%

Pediatric chronic myeloid leukemia is a unique disease that requires a different approach

Hijiya

Schultz

Metzler

et al. 2016

Blood

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163

176

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Chronic myelogenous leukemia (CML) in children is relatively rare. Because of a lack of robust clinical study evidence, management of CML in children is not standardized and often follows guidelines developed for adults. Children and young adults tend to have a more aggressive clinical presentation than older adults, and prognostic scores for adult CML do not apply to children. CML in children has been considered to have the same biology as in adults, but recent data indicate that some genetic differences exist in pediatric and adult CML. Because children with CML may receive tyrosine kinase inhibitor (TKI) therapy for many decades, and are exposed to TKIs during a period of active growth, morbidities in children with CML may be distinct from those in adults and require careful monitoring. Aggressive strategies, such as eradication of CML stem cells with limited duration and intensive regimens of chemotherapy and TKIs, may be more advantageous in children as a way to avoid lifelong exposure to TKIs and their associated adverse effects. Blood and marrow transplantation in pediatric CML is currently indicated only for recurrent progressive disease, and the acute and long-term toxicities of this option should be carefully evaluated against the complications associated with lifelong use of TKIs.

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Allogeneic stem cell transplantation for children and adolescents/young adults with de novo blastic phase chronic myeloid leukemia in the tyrosine kinase inhibitor era

Ishida

Shimada

Tanizawa³

et al. 2023

American J Hematol

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Chronic myeloid leukemia (CML) incidence increases with age, thus the disease is rare during childhood, comprising 2%-3% of childhood leukemia. 1 At diagnosis, the vast majority of children with CML present in chronic phase (CP); thus, children or adolescents/young adults (AYAs) diagnosed with blastic phase CML (de novo CML-BP) are extremely rare. 2 While tyrosine kinase inhibitors (TKIs) have significantly improved the survival of patients with CML, hematopoietic stem cell transplantation (HSCT) still plays an important role for some high-risk patients.However, few studies have investigated prognostic factors related to HSCT outcomes for children with CML in the TKI era, 3,4 and those focusing on de novo CML-BP are even more limited.Here, we investigated the characteristics and outcomes of 165 patients diagnosed with de novo CML-BP under 40 years old who received TKI treatment and their first HSCT between 2000 and 2019 in Japan. Details for methods are provided in Supplementary Methods S1.Characteristics of patients included in this study are presented in Table S1. The median follow-up period of survivors was 5.0 years after HSCT (range, 0.2-15.9 years). Comparison of the characteristics of children (0-17 years old) and AYA patients (18-39 years old) demonstrated that the former received donor-recipient sex mismatched HSCT more frequently and were more likely to undergo HSCT with better disease status. Median time from diagnosis to HSCT did not differ significantly between the two age. Among the 165 patients, 17 received reduced-intensity conditioning (RIC) before HSCT, and information on reasons for choosing a RIC regimen was available for 9 patients. The most frequent reason was patient complications present at the time of HSCT (n = 6), and the second most common reason was to avoid future long-term complications (n = 3).Five-year event-free survival (EFS) of all included patients was 64.1% (95% confidence interval [CI], 55.9%-71.2%), and 5-year overall survival (OS) was 70.3% (95% CI, 62.0%-77.1%). Cumulative incidence of relapse at 5 years was 21.2% (95% CI, 15.2%-27.9%), and cumulative incidence of non-relapse mortality at 5 years was 14.7% (95% CI, 9.5%-20.9%). These outcomes did not differ significantly between children and AYA patients as the 5-year EFS of children was 72.6% (95% CI, 55.1%-84.2%), and that of AYA patients was 61.7% (95% CI, 52.2%-69.9%; p = .226; Figure 1A), and the 5-year OS did not differ between them ( p = .507; Figure 1B). Causes of death were

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Outcomes of Allogeneic Hematopoietic Cell Transplantation in Children and Young Adults with Chronic Myeloid Leukemia: A CIBMTR Cohort Analysis

Cited by 27 publications

References 54 publications

Outcomes of allogenic hematopoietic cell transplantation for childhood chronic myeloid leukemia: Single‐center experience

Outcomes of allogenic hematopoietic cell transplantation for childhood chronic myeloid leukemia: Single‐center experience

Pediatric chronic myeloid leukemia is a unique disease that requires a different approach

Allogeneic stem cell transplantation for children and adolescents/young adults with de novo blastic phase chronic myeloid leukemia in the tyrosine kinase inhibitor era

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