Clinical Profile and Treatment Outcomes of Childhood Philadelphia-Positive Acute Lymphoblastic Leukaemia.
Source: PubMed, NCBI / U.S. National Library of Medicine
To describe the clinical presentation, treatment, and survival outcomes of paediatric Philadelphia-positive acute lymphoblastic leukaemia (Ph+ve ALL) patients. An observational study. Place and Duration of the Study: Department of Paediatric Oncology, Combined Military Hospital, and Department of Paediatric Clinical Haematology and Bone Marrow Transplant, Armed Forces Bone Marrow Transplant Centre, Rawalpindi, Pakistan, from January 2012 to June 2025. This cohort included all children aged <18 years (n = 30) diagnosed with Ph+ve ALL. The EsPhALL 2017/COG AALL1631 protocol was used for treatment. Data were collected from patient records, and descriptive statistics were calculated. Among 1,170 paediatric acute lymphoblastic leukaemia (ALL) patients, only 30 (2.56 %) had Ph+ve ALL. Twenty-six (85%) patients had de novo ALL, and four (15%) had disease transformed from chronic myeloid leukaemia. The median age was 8 ± 3.6 years, and twenty (66.7%) patients were males. Fever (86.7%), pallor (96.7%), and bruising (36.7%) were common presentations, with hepatomegaly (86.66%) and splenomegaly (83.33%) predominating. Febrile neutropenia (83.33%) and steroid-induced proximal myopathy (56.7%) were common complications during induction. Relapse occurred in nine (45%) cases -six (66.66%) involving the central nervous system (CNS) and three (33.33%) involving the bone marrow. In univariate log-rank analysis, female gender, lactate dehydrogenase (LDH), and total leukocyte count (TLC) at
Abstract
To describe the clinical presentation, treatment, and survival outcomes of paediatric Philadelphia-positive acute lymphoblastic leukaemia (Ph+ve ALL) patients. An observational study. Place and Duration of the Study: Department of Paediatric Oncology, Combined Military Hospital, and Department of Paediatric Clinical Haematology and Bone Marrow Transplant, Armed Forces Bone Marrow Transplant Centre, Rawalpindi, Pakistan, from January 2012 to June 2025. This cohort included all children aged <18 years (n = 30) diagnosed with Ph+ve ALL. The EsPhALL 2017/COG AALL1631 protocol was used for treatment. Data were collected from patient records, and descriptive statistics were calculated. Among 1,170 paediatric acute lymphoblastic leukaemia (ALL) patients, only 30 (2.56 %) had Ph+ve ALL. Twenty-six (85%) patients had de novo ALL, and four (15%) had disease transformed from chronic myeloid leukaemia. The median age was 8 ± 3.6 years, and twenty (66.7%) patients were males. Fever (86.7%), pallor (96.7%), and bruising (36.7%) were common presentations, with hepatomegaly (86.66%) and splenomegaly (83.33%) predominating. Febrile neutropenia (83.33%) and steroid-induced proximal myopathy (56.7%) were common complications during induction. Relapse occurred in nine (45%) cases -six (66.66%) involving the central nervous system (CNS) and three (33.33%) involving the bone marrow. In univariate log-rank analysis, female gender, lactate dehydrogenase (LDH), and total leukocyte count (TLC) at the time of admission affected disease-free survival (DFS) at 5 years (p <0.05), while female gender and LDH were significant for 5-year overall survival (OS; p <0.05). OS was 43%, with a mean survival of 1,931.96 days (CI 95%: 1247.97-2615.95), and DFS was 33.3%, with a mean survival of 678.1 days (CI 95%: 430.42-926.59). Despite TKI availability, Ph+ve ALL outcomes were inferior to trial data, with high rates of CNS relapse, underscoring the need for locally adapted protocols with improved CNS prophylaxis, including triple immunotherapy. Ph+ve ALL, central nervous system relapse, Pakistan, Paediatric leukaemia, Survival outcomes.