<scp>HLA</scp>‐matched related donor hematopoietic stem cell transplantation is a suitable treatment in adolescents and adults with sickle cell disease: Comparison of myeloablative and non‐myeloablative approaches

Dhédin, Nathalie; Chevillon, Florian; Castelle, Martin; Lavoipière, Virginie; Vasseur, Loïc; Dalle, Jean‐Hugues; Joseph, Laure; Beckerich, Florence; Buchbinder, Nimrod; Coman, Téreza; Garban, Frédéric; Ferster, Alina; Nguyen, Stéphanie; Boissel, Nicolas; Arlet, Jean‐Benoît; Pondarré, Corinne

doi:10.1002/ajh.26656

Cited by 2 publications

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“…In a study by Gluckman et al., of which 873(87.0%) patients received MAC and 125(13%) transplantations used different types of RIC, the type of conditioning regimen intensity had no significant effect on EFS or on OS 13 . Interestingly, non‐myeloablative (NMA) TBI‐based regimen developed by NIH group was successful in inducing durable donor chimerism in two pediatric SCD studies similar to reports in adolescents and adults 21–25 . The variations in the outcomes of transplantations using RIC/NMA regimens require further studies in light of its potential benefits in minimizing the risks of sterility and growth retardation when compared to MAC regimen.…”

Section: Discussionmentioning

confidence: 85%

“…13 Interestingly, non-myeloablative (NMA) TBI-based regimen developed by NIH group was successful in inducing durable donor chimerism in two pediatric SCD studies similar to reports in adolescents and adults. [21][22][23][24][25] The variations in the outcomes of transplantations using RIC/NMA regimens require further studies in light of its potential benefits in minimizing the risks of sterility and growth retardation when compared to MAC regimen. Two of our patients treated with RIC did well with full donor chimerism.…”

Section: Discussionmentioning

confidence: 99%

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The effect of intensity of conditioning regimen on the outcome of HSCT in children with sickle cell disease

Alsultan

Abujoub

Elbashir

et al. 2022

Clinical Transplantation

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Background: Allogeneic hematopoietic stem cell transplantation (HSCT) provides a cure for patients with sickle cell disease (SCD). This study describes the effect of conditioning regimen intensity on HSCT outcomes among children younger than 14 years with SCD. Methods:Transplants from HLA-matched related donors (MRD) and unrelated donors (MUD) using either myeloablative conditioning (MAC) regimens or reduced intensity conditioning (RIC) regimens were considered. Event-free survival (EFS) was the primary endpoint. Secondary endpoints included overall survival (OS) and occurrence of GVHD.Results: 48 SCD patients underwent HSCT, 45 (93.8%) patients had MRD, 1 (2.1%) had 9/10 related donor, and 2 (4.1%) had MUD. The median age at transplant was 8.6 years (range, 3.1-13.8). Conditioning regimens were myeloablative (MAC) in 41 (85.4%) patients and of reduced intensity in 7 (14.6%) patients. EFS at 2 years was 100% among MAC group compared to 29% in the RIC group (p < .001). The median follow-up was 43.4 months (range 26.8-134). All events in the RIC group were secondary graft failure. However, OS was 100% in both groups at 2 years. Acute GVHD II-IV was diagnosed in 2 (4.1%) patients. Chronic GVHD occurred in 2 (4.1%) patients.GVHD did not occur in patients who underwent MUD HSCT.Conclusions: MAC in children with SCD is well tolerated and associated with an excellent outcome for HLA-matched HSCT in SCD. There was a high rate of secondary graft failure with the use of RIC. Future studies are needed to optimize RIC regimens in HSCT of children with SCD.

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Section: Discussionmentioning

confidence: 85%

Section: Discussionmentioning

confidence: 99%

The effect of intensity of conditioning regimen on the outcome of HSCT in children with sickle cell disease

Alsultan

Abujoub

Elbashir

et al. 2022

Clinical Transplantation

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Alloimmunization and hyperhemolysis in sickle cell disease

Pirenne,

Pondarré

2023

Hematology

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Alloimmunization against red blood cell antigens and delayed hemolytic transfusion reaction (DHTR) are major barriers to transfusion in sickle cell disease (SCD). In SCD, DHTR is a potentially life-threatening. Blood group polymorphism in SCD patients, who are of African ancestry and frequently exposed to antigens they do not carry; an inflammatory clinical state; and occasional transfusion in acute situations are risk factors for alloimmunization and DHTR. In patients at risk, the transfusion indication must be balanced against the risk of developing DHTR. However, when transfusion is absolutely necessary, protocols combining the prevention of exposure to immunogenic antigens with immunosuppressive treatments must be implemented, and patients should be carefully monitored during posttransfusion follow-up. This close monitoring makes it possible to diagnose hyperhemolysis as soon as possible; to avoid retransfusion, which can exacerbate hemolysis; and to administer specific treatments, such as anticomplement therapy, in severe cases. Finally, in patients with severe disease, hematopoietic stem cell transplantation may be indicated. However, transfusion is also required in this context, and its management is complex because these risks must be taken into account.

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HLA‐matched related donor hematopoietic stem cell transplantation is a suitable treatment in adolescents and adults with sickle cell disease: Comparison of myeloablative and non‐myeloablative approaches

Cited by 2 publications

References 6 publications

The effect of intensity of conditioning regimen on the outcome of HSCT in children with sickle cell disease

The effect of intensity of conditioning regimen on the outcome of HSCT in children with sickle cell disease

Alloimmunization and hyperhemolysis in sickle cell disease

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