Haploidentical Allogeneic Stem Cell Transplantation in Sickle Cell Disease: A Systematic Review and Meta-Analysis

Aydin, Mesire; Dovern, Elisabeth; Leeflang, Mariska; Fuente, Josu de la; Kassim, Adetola A.; Biemond, Bart J.; Nur, Erfan

doi:10.1016/j.jtct.2021.09.009

Cited by 26 publications

(18 citation statements)

References 30 publications

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“…In a meta-analysis, patients having sickle cell disease who underwent haploidentical transplantation using T-cell depletion and PTCy were compared [14]. The meta-analysis included only two studies that used T-cell depletion methods [15,16].…”

Section: Discussionmentioning

confidence: 99%

Comparison of alpha/beta T cell depletion with posttransplant cyclophosphamide in haploidentical transplantation

Ozen¹,

Gündüz²,

Aksoyoglu³

et al. 2023

j-ebr

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To compare alpha/beta T-cell depletion with posttransplant Cyclophosphamide (PTCy) in haploidentical allogeneic transplantation in adult hematological patients, this is the first study. Method: In our study, we reported 36 haploidentical allogeneic stem cell transplants which were performed in our clinic. Results: Twenty-six of these haploidentical transplants received standard treatment and transplanted either with PTCy (n=21, 81%) or with alpha/beta T-cell depletion (n=5, 19%). Less CD34+ stem cells were administered in the T-cell depletion group. When the two groups were compared in terms of survival, no difference was found in relapse-free and overall survival in each group. Acute GVHD cases developed in the PTCy group mostly developed after CMV infection, whereas acute GVHD did not develop in the T-cell depletion group. In the PTCy group, 8 cases developed graft failure and relapse; 4 cases developed graft failure or relapse in the T-cell depletion group, and 2 of them developed graft failure or relapse following EBV infection and 1 of them following CMV infection. Conclusion:We have a small number of patients in the T cell depletion group. Due to our long follow-up period, we believe that our patients with T cell depletion can be compared with those who underwent PTCy, and similar survival results can be achieved in adult patients having hematologic malignancies.

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Section: Discussionmentioning

confidence: 99%

Comparison of alpha/beta T cell depletion with posttransplant cyclophosphamide in haploidentical transplantation

Ozen¹,

Gündüz²,

Aksoyoglu³

et al. 2023

j-ebr

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“…Other hematological settings where such alemtuzumab-based conditioning combined with very low-dose chemotherapy may lead to the cure of disease through the long term establishment of mixed donor chimerism, 12,13 may help in clarifying the respective roles of diseasespecific and/or transplant-specific determinants of transplant engraftment. As safety of haploidentical HSCT in SCD patients has improved in recent years, 14 the impact of ABO matching on outcome in this setting will also be a matter of study in the near future.…”

Section: Discussionmentioning

confidence: 99%

ABO blood barrier to engraftment after allogeneic stem cell transplantation in sickle cell disease: A case‐story with two successive HLA‐matched sibling donors

Redjoul¹,

Beckerich²,

Luna

et al. 2022

American J Hematol

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“…Stem cells are usually harvested from the bone marrow, peripheral blood, or cord blood [ 181 ]. Although less than 14% of individuals with SCA have HLA-matched siblings as potential donors [ 46 ], available data shows that HLA-haploidentical (half-matched) stem cell transplant is a viable alternative, with patients having a 91% overall survival and low transplantation-related toxicity [ 182 ], comparable with 92.9% (95% confidence interval: 91.1%-94.6%) overall survival among HLA-matched sibling HSCT [ 181 ]. This has been made possible by improvements in conditioning regimens, robust pre- and post-transplantation T-cell depletion, and improvement in supportive care [ 182 ].…”

Section: Comprehensive Care Of Children With Scdmentioning

confidence: 99%

Sickle Cell Disease in Children and Adolescents: A Review of the Historical, Clinical, and Public Health Perspective of Sub-Saharan Africa and Beyond

Egesa

Nakalema

Waibi

et al. 2022

International Journal of Pediatrics

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Sickle cell disease (SCD) is an umbrella term for a group of life-long debilitating autosomal recessive disorders that are caused by a single-point mutation (Glu→Val) that results in polymerization of hemoglobin (Hb) and reversible sickle-shape deformation of erythrocytes. This leads to increased hemolysis of erythrocytes and microvascular occlusion, ischemia-reperfusion injury, and tissue infarction, ultimately causing multisystem end-organ complications. Sickle cell anemia (HbSS) is the most common and most severe genotype of SCD, followed by HbSC, HbSβ0thalassemia, HbSβ+thalassemia, and rare and benign genotypes. Clinical manifestations of SCD occur early in life, are variable, and are modified by several genetic and environmental factors. Nearly 500 children with SCD continue to die prematurely every day, due to delayed diagnosis and/or lack of access to comprehensive care in sub-Saharan Africa (SSA), a trend that needs to be urgently reversed. Despite proven efficacy in developed countries, newborn screening programs are not universal in SSA. This calls for a consolidated effort to make this possible, through the use of rapid, accurate, and cheap point-of-care test kits which require minimal training. For almost two decades, hydroxyurea (hydroxycarbamide), a century-old drug, was the only disease-modifying therapy approved by the U.S. Food and Drug Administration. Recently, the list expanded to L-glutamine, crizanlizumab, and voxelotor, with several promising novel therapies in the pipeline. Despite its several limitations, hematopoietic stem cell transplant (HSCT) remains the only curative intervention for SCD. Meanwhile, recent advances in gene therapy trials offer a glimpse of hope for the near future, although its use maybe limited to developed countries for several decades.

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Haploidentical Allogeneic Stem Cell Transplantation in Sickle Cell Disease: A Systematic Review and Meta-Analysis

Cited by 26 publications

References 30 publications

Comparison of alpha/beta T cell depletion with posttransplant cyclophosphamide in haploidentical transplantation

Comparison of alpha/beta T cell depletion with posttransplant cyclophosphamide in haploidentical transplantation

ABO blood barrier to engraftment after allogeneic stem cell transplantation in sickle cell disease: A case‐story with two successive HLA‐matched sibling donors

Sickle Cell Disease in Children and Adolescents: A Review of the Historical, Clinical, and Public Health Perspective of Sub-Saharan Africa and Beyond

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