Real-world Experience of Imatinib in Pediatric Chronic Phase Chronic Myeloid Leukemia: A Single-center Experience From India

Ganguly, Shuvadeep; Pushpam, Deepam; Mian, Agrima; Chopra, Anita; Gupta, Ritu; Bakhshi, Sameer

doi:10.1016/j.clml.2020.02.015

Cited by 6 publications

(9 citation statements)

References 40 publications

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“…Similar to our experience, 20% of children presented with advanced disease in a study by Ganguly et al from another major cancer hospital in India. 22 A study from Japan reported that around 10% (23/238) of children with CML (including both chronic and advanced phases) had clinical leucostasis in their patients, which was twice as common compared to our study (5.7%). Furthermore, ocular symptoms were the most frequently observed (14/238) symptom in the Japanese study, followed by priapism (4/238), whereas pulmonary leucostasis was more common in our cohort.…”

Section: Discussioncontrasting

confidence: 54%

“…23 The mean (SD) time to CCyR and MMR in our cohort was significantly delayed when compared to the milestones suggested in the modified ELN/NCCN guidelines. Data from the published literature 11,[19][20][21][22] on cytogenetic and molecular response in children are quite varied (Table 4). Despite the delay in response to imatinib, only three patients in the entire cohort had progressed to BC and excellent EFS and OS were observed in this cohort similar to other studies from India.…”

Section: Discussionmentioning

confidence: 99%

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Treatment and follow‐up of children with chronic myeloid leukaemia in chronic phase (CML‐CP) in the tyrosine kinase inhibitor (TKI) era—Two decades of experience from the Tata Memorial Hospital paediatric CML (pCML) cohort

Roy Moulik,

Keerthivasagam,

Pandey

et al. 2023

Br J Haematol

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SummaryTyrosine kinase inhibitors (TKIs) have drastically improved the outcomes of pCML (paediatric CML) but data on long‐term off‐target toxicities of TKIs in children are scarce. In this single‐centre, retrospective cum prospective study of pCML in chronic phase, we report our experience of treating 173 children with imatinib and following them for long‐term toxicities. Mean (SD) time to attain CHR, CCyR and MMR were 3.05 (2.1), 10.6 (8.4) and 43.4 (31.8) months respectively. DMR was not attained in 59 (34%) patients at last follow‐up. Ten patients were switched to second‐generation TKIs (2G‐TKIs; nilotinib = 1/dasatinib = 9) due to poor/loss in response, of which seven had kinase domain mutations. Three patients progressed to the blastic phase. At a median follow‐up of 84 (3–261) months, the 5‐year EFS and OS for the entire cohort were 96.9% (95% CI: 93.4–100) and 98.7% (95% CI: 96.9–100) respectively. Screening for long‐term toxicities revealed low bone density and hypovitaminosis D in 70% and 80% respectively. Other late effects included short stature (27%), delayed puberty (15%), poor sperm quality (43%) and miscellaneous endocrinopathies (8%). Children younger than 5 years at diagnosis were more susceptible to growth and endocrine toxicities (p = 0.009). Regular monitoring for long‐term toxicities, timely intervention and trial of discontinuation whenever feasible are likely to improve the long‐term outlook of pCML.

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Section: Discussioncontrasting

confidence: 54%

Section: Discussionmentioning

confidence: 99%

Treatment and follow‐up of children with chronic myeloid leukaemia in chronic phase (CML‐CP) in the tyrosine kinase inhibitor (TKI) era—Two decades of experience from the Tata Memorial Hospital paediatric CML (pCML) cohort

Roy Moulik,

Keerthivasagam,

Pandey

et al. 2023

Br J Haematol

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“…There is a 35% gap between EFS and OS in our study, which is comparable to the observation of a 28% gap in another report (5-year EFS and OS: 64% and 92% respectively). 20 This gap highlights the fact that many patients with suboptimal responses to imatinib continue to respond for a prolonged time and the lower EFS is mostly due to noncompliance to treatment in our study. More recently, the availability of second-generation TKIs like nilotinib and dasatinib can be prescribed to those who fail imatinib.…”

Section: Discussionmentioning

confidence: 78%

“…In a report from Delhi, 3% and 17% of patients had a diagnosis of AP and BC respectively. 20 Reports from developed countries indicate that 2-6% of children present in AP and 2-4% BC. 6,21 CML in children is different compared with adults ($2% AP and 1% BC), as a higher proportion present in AP and BC.…”

Section: Discussionmentioning

confidence: 99%

“…5,[23][24][25][26][27][28] We observed a male to female ratio of 1.3:1-this is similar to reports from southern India and considerably lesser than a higher male to female ratio reported by studies from northern and western India. 20,[23][24][25][26][27][28] The higher male to female ratio in CML observed in northwestern India could be due to social bias where females are not provided similar access to healthcare as males.…”

Section: Discussionmentioning

confidence: 99%

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Real-World Experience of Treating Pediatric Chronic Myeloid Leukemia: Retrospective Study from a Cancer Center in Southern India

Kesana

Radhakrishnan

Kalaiyarasi

et al. 2021

Indian J Med Paediatr Oncol

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Introduction Chronic myeloid leukemia (CML) is rare in children and constitutes 2% of all leukemia. We present our institute experience in treating pediatric CML for 20 years. Objectives There is a paucity of data on pediatric CML from India, hence we would like to present treatment responses and survival rates in our pediatric population treated with tyrosine kinase inhibitors at our center. Materials and Methods Patients aged less than 18 years, diagnosed with CML from 2000 to 2019, and treated with imatinib were analyzed retrospectively considering demographic features, treatment characteristics, and survival outcomes. Descriptive analysis was done for the baseline characteristics. Event-free survival (EFS) and overall survival (OS) were calculated using the Kaplan-Meier method and the factors were compared using the log-rank test. Results During the study period, 95 patients were diagnosed with CML of which 54 (56.8%) were males. The most common stage at presentation was the chronic phase (CP) with 84 (88.4%) patients followed by accelerated phase (AP) and blast crisis (BC) with 6 (6.3%) and 5 (5.3%) patients respectively. The median duration of follow-up for all patients was 98 months. EFS and OS at 8 years for patients with CML-CP were 43.1% and 80.4% respectively. Complete hematological response, complete cytogenetic response, and major molecular response was documented in 91 (95.7%), 73 (76.8%), and 63 (66.3%) patients respectively. Conclusion Outcomes in pediatric CML are comparable to that of adults. Imatinib is well tolerated in children.

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Philadelphia Chromosome Positive and Philadelphia-Like Acute Lymphoblastic Leukemia in Children and Adolescents: Current Management, Controversies and Emerging Concepts

Ganguly,

Sasi,

Pushpam

et al. 2023

Indian J Pediatr

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Real-world Experience of Imatinib in Pediatric Chronic Phase Chronic Myeloid Leukemia: A Single-center Experience From India

Cited by 6 publications

References 40 publications

Treatment and follow‐up of children with chronic myeloid leukaemia in chronic phase (CML‐CP) in the tyrosine kinase inhibitor (TKI) era—Two decades of experience from the Tata Memorial Hospital paediatric CML (pCML) cohort

Treatment and follow‐up of children with chronic myeloid leukaemia in chronic phase (CML‐CP) in the tyrosine kinase inhibitor (TKI) era—Two decades of experience from the Tata Memorial Hospital paediatric CML (pCML) cohort

Real-World Experience of Treating Pediatric Chronic Myeloid Leukemia: Retrospective Study from a Cancer Center in Southern India

Philadelphia Chromosome Positive and Philadelphia-Like Acute Lymphoblastic Leukemia in Children and Adolescents: Current Management, Controversies and Emerging Concepts

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