The role of HLA matching in unrelated donor hematopoietic stem cell transplantation for sickle cell disease in Europe

Gluckman, Éliane; Fuente, Josu de la; Cappelli, Barbara; Scigliuolo, Graziana Maria; Volt, Fernanda; Tozatto-Maio, Karina; Rocha, Vanderson; Mina, Tommaso; O'Boyle, Farah; Smiers, Frans J.; Cunha-Riehm, Claudia Bettoni Da; Calore, Elisabetta; Bonanomi, Sonia; Graphakos, Stelios; Paisiou, Anna; Albert, Michael H.; Ruggeri, Annalisa; Zecca, Marco; Lankester, Arjan C.; Corbacioglu, Selim

doi:10.1038/s41409-020-0847-z

Cited by 14 publications

(11 citation statements)

References 31 publications

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“…None of them presented with SCD‐related symptoms so far (Table 4). The absence of mixed donor chimerisms in patients receiving grafts from unrelated donors can be explained by the stronger alloreactive potential present in unrelated donor transplantations 26,27 . A higher risk for acute GvHD is associated with alloreactivity requiring an intensive GvHD prophylaxis.…”

Section: Discussionmentioning

confidence: 99%

Allogeneic hematopoietic stem cell transplantation from sibling and unrelated donors in pediatric patients with sickle cell disease—A single center experience

Kogel

Hakimeh

Sodani

et al. 2020

Pediatric Transplantation

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HSCT is curative in SCD. Patients with HLA‐identical sibling donor have an excellent outcome ranging from 90%‐100% overall and event‐free survival. However, due to the lack of matched sibling donors this option is out of reach for 70% of patients with SCD. The pool of potential donors needs to be extended. Transplantations from HLA‐matched unrelated donors were reported to be less successful with shorter event‐free survival and higher incidences of complications including graft‐vs‐host disease, especially in patients with advanced stage SCD. Here we report transplantation outcomes for 25 children with SCD transplanted using HLA‐matched grafts from related or unrelated donors. Overall survival was 100% with no severe (grade III‐IV) graft‐vs‐host disease and a 12% rejection rate. Mixed donor chimerisms only occurred in transplantations from siblings, while transplantations from unrelated donors resulted in either complete donor chimerism or rejection. Despite the small patient number, overall and disease‐free survival for unrelated donor transplantations is excellent in this cohort. The advanced disease state, higher alloreactive effect and stronger immunosuppression in unrelated donor transplantations raises patient risk, for which possible solutions could be found in optimization of transplant preparation, graft manipulation or haploidentical transplantation using T cell receptor α/β‐depleted grafts.

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Section: Discussionmentioning

confidence: 99%

Allogeneic hematopoietic stem cell transplantation from sibling and unrelated donors in pediatric patients with sickle cell disease—A single center experience

Kogel

Hakimeh

Sodani

et al. 2020

Pediatric Transplantation

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“…As a consequence, many of these unrelated allo-HSTC were performed with some level of HLA mismatch and were associated with a high prevalence of engraftment failure as well as graft-versus-host disease ( 53 , 54 ). Recently, important advances have been reported, both with improved match between unrelated patients and donors (HLA high resolution full match or haploidentical) and in treatment with post-transplant drugs ( 55 ). However, these studies are not sufficient to guarantee the safety of this treatment ( 52 ), which is still evaluated as experimental.…”

Section: Discussionmentioning

confidence: 99%

How Ancestry Influences the Chances of Finding Unrelated Donors: An Investigation in Admixed Brazilians

et al. 2020

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associated with reduced chances of finding a match (up to 60% reduction). Finally, we document that the strongest reduction in chances of finding a match is associated with having an MHC region of exclusively African ancestry (up to 75% reduction). We apply our findings to a specific condition, for which there is a clinical indication for transplantation: sickle-cell disease. We show that the increased African ancestry in patients with this disease leads to reduced chances of finding a match, when compared to the remainder of the sample, without the condition. Our results underscore the influence of ancestry on chances of finding compatible HLA matches, and indicate that efforts guided to increasing the African component of registries are necessary.

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“…Aber auch die Studien mit Fremdspendern und haploidenten Spendern (in der Regel ein Elternteil) liefern sehr vielversprechende und ähnlich gute Resultate. Transplantationen mit Zellen von alternativen Spendern sollten jedoch nur im Rahmen von Studien durchgeführt werden 15 – 18 .…”

Section: Therapieunclassified

Neugeborenenscreening auf Sichelzellkrankheit

Lobitz¹

2022

Kinder- und Jugendmedizin

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ZUSAMMENFASSUNGDie Sichelzellkrankheit (SCD) ist eine schwere angeborene Bluterkrankung, die mit einer hohen Morbidität und Mortalität im Säuglings- und Kleinkindalter einhergeht, wenn sie noch nicht diagnostiziert worden ist. Kritisch sind insbesondere fulminante bakterielle Infektionen im Rahmen der krankheitstypischen Hyposplenie sowie akute Verschlechterungen der im Intervall stabilen Anämie. Mit sehr einfachen Maßnahmen (Schulung, Impfung und Penicillin-Prophylaxe) lässt sich ein Großteil der schweren Komplikationen vermeiden. Da die Erkrankung schon bei Geburt sehr einfach und sehr zuverlässig aus Trockenblut diagnostizierbar ist und zudem inzwischen ein gutes therapeutisches Konzept existiert, gibt es eine gute Rationale für ein Neugeborenenscreening (NBS). International werden Neugeborene teilweise schon seit den frühen 1970er-Jahren auf die SCD untersucht. Das flächendeckende NBS in Deutschland hat am 1. Oktober 2021 begonnen. Dieser Artikel gibt Ihnen einen Überblick über die neue Zielkrankheit.

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The role of HLA matching in unrelated donor hematopoietic stem cell transplantation for sickle cell disease in Europe

Cited by 14 publications

References 31 publications

Allogeneic hematopoietic stem cell transplantation from sibling and unrelated donors in pediatric patients with sickle cell disease—A single center experience

Allogeneic hematopoietic stem cell transplantation from sibling and unrelated donors in pediatric patients with sickle cell disease—A single center experience

How Ancestry Influences the Chances of Finding Unrelated Donors: An Investigation in Admixed Brazilians

Neugeborenenscreening auf Sichelzellkrankheit

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