{"title":"婴儿急性淋巴细胞白血病:来自印度的观点","authors":"Debasish Sahoo, Sonali Mohapatra","doi":"10.1016/j.phoj.2025.100467","DOIUrl":null,"url":null,"abstract":"<div><div>Improvement in outcomes of pediatric acute lymphoblastic leukemia (ALL) has been a success story with survival approaching 90% in various studies. However, ALL in infants, though rare, is an exception, with its distinct aggressive disease biology, molecular genetics and poor response to therapy with an excess of relapses. It characteristically presents with hyperleukocytosis, bulky disease and a higher incidence of central nervous system (CNS) and cutaneous involvement. Immunophenotyping is notable for CD10 negativity, and higher co-expression of various myeloid markers indicating a primitive hematopoietic cell of origin. Rearrangement in <em>KMT2A</em> gene i.e. <em>KMTA</em>-r (previously <em>MLL</em>), located on 11q23, is central to disease pathophysiology, playing a key role in epigenetic modification that drives leukemogenesis, and is the strongest negative prognostic marker in this subgroup. Hyperleukocytosis with total leukocyte count exceeding 3,00,000/mm<sup>3</sup>, younger age group (<6 months age), poor prednisolone response and poor MRD response are other important negative prognostic markers in infant ALL. Despite highly intensive therapy and hematopoietic stem cell transplantation (HSCT), the incidence of relapse remains exceedingly high. Significant treatment related mortality and morbidity remain other barriers to successful outcomes. Recently, the use of blinatumomab has shown promising results in infant ALL and is being integrated into chemotherapy backbones in various trials e.g. Interfant 06. Early integration of immunotherapy including blinatumomab and chimeric antigen receptor (CAR) T cells, and other novel drugs in larger collaborative trials will be key in improving the prognosis in this population.</div></div>","PeriodicalId":101004,"journal":{"name":"Pediatric Hematology Oncology Journal","volume":"10 3","pages":"Article 100467"},"PeriodicalIF":0.0000,"publicationDate":"2025-06-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Infant acute lymphoblastic leukemia: a view from India\",\"authors\":\"Debasish Sahoo, Sonali Mohapatra\",\"doi\":\"10.1016/j.phoj.2025.100467\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<div><div>Improvement in outcomes of pediatric acute lymphoblastic leukemia (ALL) has been a success story with survival approaching 90% in various studies. However, ALL in infants, though rare, is an exception, with its distinct aggressive disease biology, molecular genetics and poor response to therapy with an excess of relapses. It characteristically presents with hyperleukocytosis, bulky disease and a higher incidence of central nervous system (CNS) and cutaneous involvement. Immunophenotyping is notable for CD10 negativity, and higher co-expression of various myeloid markers indicating a primitive hematopoietic cell of origin. Rearrangement in <em>KMT2A</em> gene i.e. <em>KMTA</em>-r (previously <em>MLL</em>), located on 11q23, is central to disease pathophysiology, playing a key role in epigenetic modification that drives leukemogenesis, and is the strongest negative prognostic marker in this subgroup. Hyperleukocytosis with total leukocyte count exceeding 3,00,000/mm<sup>3</sup>, younger age group (<6 months age), poor prednisolone response and poor MRD response are other important negative prognostic markers in infant ALL. Despite highly intensive therapy and hematopoietic stem cell transplantation (HSCT), the incidence of relapse remains exceedingly high. Significant treatment related mortality and morbidity remain other barriers to successful outcomes. Recently, the use of blinatumomab has shown promising results in infant ALL and is being integrated into chemotherapy backbones in various trials e.g. Interfant 06. Early integration of immunotherapy including blinatumomab and chimeric antigen receptor (CAR) T cells, and other novel drugs in larger collaborative trials will be key in improving the prognosis in this population.</div></div>\",\"PeriodicalId\":101004,\"journal\":{\"name\":\"Pediatric Hematology Oncology Journal\",\"volume\":\"10 3\",\"pages\":\"Article 100467\"},\"PeriodicalIF\":0.0000,\"publicationDate\":\"2025-06-14\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Pediatric Hematology Oncology Journal\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://www.sciencedirect.com/science/article/pii/S246812452500035X\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"\",\"JCRName\":\"\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Pediatric Hematology Oncology Journal","FirstCategoryId":"1085","ListUrlMain":"https://www.sciencedirect.com/science/article/pii/S246812452500035X","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"","JCRName":"","Score":null,"Total":0}
Infant acute lymphoblastic leukemia: a view from India
Improvement in outcomes of pediatric acute lymphoblastic leukemia (ALL) has been a success story with survival approaching 90% in various studies. However, ALL in infants, though rare, is an exception, with its distinct aggressive disease biology, molecular genetics and poor response to therapy with an excess of relapses. It characteristically presents with hyperleukocytosis, bulky disease and a higher incidence of central nervous system (CNS) and cutaneous involvement. Immunophenotyping is notable for CD10 negativity, and higher co-expression of various myeloid markers indicating a primitive hematopoietic cell of origin. Rearrangement in KMT2A gene i.e. KMTA-r (previously MLL), located on 11q23, is central to disease pathophysiology, playing a key role in epigenetic modification that drives leukemogenesis, and is the strongest negative prognostic marker in this subgroup. Hyperleukocytosis with total leukocyte count exceeding 3,00,000/mm3, younger age group (<6 months age), poor prednisolone response and poor MRD response are other important negative prognostic markers in infant ALL. Despite highly intensive therapy and hematopoietic stem cell transplantation (HSCT), the incidence of relapse remains exceedingly high. Significant treatment related mortality and morbidity remain other barriers to successful outcomes. Recently, the use of blinatumomab has shown promising results in infant ALL and is being integrated into chemotherapy backbones in various trials e.g. Interfant 06. Early integration of immunotherapy including blinatumomab and chimeric antigen receptor (CAR) T cells, and other novel drugs in larger collaborative trials will be key in improving the prognosis in this population.