Allogeneic hematopoietic cell transplantation (HCT) can halt organ damage and eliminate symptoms in hemoglobin disorders, including sickle cell disease (SCD) and thalassemia major. Managing the residual manifestations of pre-HCT disease complications and the long-term effects of HCT requires systematic monitoring, follow-up and intervention when indicated. Late complications vary with age and disease status at HCT and with transplant variables such as preparative regimen, donor source and compatibility, and immune reconstitution. An international consensus conference sponsored by the Pediatric Blood and Marrow Transplant Consortium in May 2016 entitled “Late Effects Screening and Recommendations Following HCT for Immune Deficiency and Nonmalignant Hematologic Disorders” focused on follow-up after HCT for hemoglobinopathy. An earlier publication from experts who participated in this session described the pathophysiology and spectrum of complications that HCT recipients experience after HCT for SCD and thalassemia major. This companion publication summarizes the consensus reached by this group of experts about long-term follow-up guidelines after HCT for hemoglobinopathy. In addition, these guidelines might also be included in studies of novel curative therapies such as autologous HCT after hematopoietic progenitor stem cell gene modification.
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Financial disclosure: This work was supported in part by grants from the Children's Discovery Institute of Washington University and St. Louis Children's Hospital (to S.S.), the National Heart, Lung, and Blood Institute and the National Cancer Institute ( U10-HL069294 to the Blood and Marrow Transplant Clinical Trials Network and the Pediatric Blood and Marrow Transplant Consortium, M.A.P.), and the National Institutes of Health grants 1R13CA159788-01 (to M.A.P. and K.S.B.) and RO1 CA07893 (to K.S.B.).
- Late effects
- Sickle cell disease