Subsequent neoplasms and late mortality in children undergoing allogeneic transplantation for nonmalignant diseases
Open Access
- 11 May 2020
- journal article
- research article
- Published by American Society of Hematology in Blood Advances
- Vol. 4 (9), 2084-2094
- https://doi.org/10.1182/bloodadvances.2019000839
Abstract
We examined the risk of subsequent neoplasms (SNs) and late mortality in children and adolescents undergoing allogeneic hematopoietic cell transplantation (HCT) for nonmalignant diseases (NMDs). We included 6028 patients (median age, 6 years; interquartile range, 1-11; range, <1 to 20) from the Center for International Blood and Marrow Transplant Research (1995-2012) registry. Standardized mortality ratios (SMRs) in 2-year survivors and standardized incidence ratios (SIRs) were calculated to compare mortality and SN rates with expected rates in the general population. Median follow-up of survivors was 7.8 years. Diagnoses included severe aplastic anemia (SAA; 24%), Fanconi anemia (FA; 10%), other marrow failure (6%), hemoglobinopathy (15%), immunodeficiency (23%), and metabolic/leukodystrophy syndrome (22%). Ten-year survival was 93% (95% confidence interval [95% CI], 92% to 94%; SMR, 4.2; 95% CI, 3.7-4.8). Seventy-one patients developed SNs (1.2%). Incidence was highest in FA (5.5%), SAA (1.1%), and other marrow failure syndromes (1.7%); for other NMDs, incidence was <1%. Hematologic (27%), oropharyngeal (25%), and skin cancers (13%) were most common. Leukemia risk was highest in the first 5 years posttransplantation; oropharyngeal, skin, liver, and thyroid tumors primarily occurred after 5 years. Despite a low number of SNs, patients had an 11-fold increased SN risk (SIR, 11; 95% CI, 8.9-13.9) compared with the general population. We report excellent long-term survival and low SN incidence in an international cohort of children undergoing HCT for NMDs. The risk of SN development was highest in patients with FA and marrow failure syndromes, highlighting the need for long-term posttransplantation surveillance in this population.This publication has 32 references indexed in Scilit:
- Malignancies after Hematopoietic Cell Transplantation for Primary Immune Deficiencies: A Report from the Center for International Blood and Marrow Transplant ResearchTransplantation and Cellular Therapy, 2011
- Allogeneic hematopoietic stem cell transplantation for sickle cell disease: the time is nowBlood, 2011
- Long-Term Survival and Late Deaths After Allogeneic Hematopoietic Cell TransplantationJournal of Clinical Oncology, 2011
- Secondary solid cancers after allogeneic hematopoietic cell transplantation using busulfan-cyclophosphamide conditioningBlood, 2011
- Long-term Clinical Outcome of Patients with Severe Combined Immunodeficiency Who Received Related Donor Bone Marrow Transplants without Pretransplant Chemotherapy or Post-transplant GVHD ProphylaxisThe Journal of Pediatrics, 2009
- Solid cancers after allogeneic hematopoietic cell transplantationBlood, 2009
- Late mortality after allogeneic hematopoietic cell transplantation and functional status of long-term survivors: report from the Bone Marrow Transplant Survivor StudyBlood, 2007
- Outcome of patients with acquired aplastic anemia given first line bone marrow transplantation or immunosuppressive treatment in the last decade: a report from the European Group for Blood and Marrow TransplantationHaematologica, 2007
- Risk of head and neck squamous cell cancer and death in patients with Fanconi anemia who did and did not receive transplantsBlood, 2005
- Solid Cancers after Bone Marrow TransplantationThe New England Journal of Medicine, 1997