Ali D Alahmari, Saad Alghamdi, Reem Alasbali, Sara Hisham Samarkandi, Saleh A Alqahtani, Hadeel Samarkandi, Syed Osman Ahmed, Dieter Broering, Hazzaa Alzahrani, Adetola Kassim, Mahmoud Aljurf, Fahad Almohareb, Waleed Al-Hamoudi
{"title":"Haplo-stem cell transplant post liver transplantation to cure sickle cell disease with related liver dysfunction: a case series.","authors":"Ali D Alahmari, Saad Alghamdi, Reem Alasbali, Sara Hisham Samarkandi, Saleh A Alqahtani, Hadeel Samarkandi, Syed Osman Ahmed, Dieter Broering, Hazzaa Alzahrani, Adetola Kassim, Mahmoud Aljurf, Fahad Almohareb, Waleed Al-Hamoudi","doi":"10.1038/s43856-025-01040-0","DOIUrl":null,"url":null,"abstract":"<p><strong>Background: </strong>Sickle cell disease (SCD) is debilitating, with age-dependent complications such as stroke and liver disease, leading to significant morbidity and early mortality in young adults. Hematopoietic stem cell transplantation (HCT) is curative treatment for SCD, but transplantation-related risks often deter its use, especially in patients with severe comorbidities. A subset of severe SCD patients with significant end-organ dysfunction may benefit from a combined approach of HCT and solid organ transplantation (SOT).</p><p><strong>Methods: </strong>To the best of our knowledge, this report presents, for the first time, the utilization of dual orthotopic liver transplantation and haploidentical HCT (haplo-HCT) for severe SCD with advanced liver cirrhosis. Employing nonmyeloablative conditioning regimen and post-transplantation cyclophosphamide (PTCy).</p><p><strong>Results: </strong>Both patients undergo orthotopic liver transplantation followed by haplo-HCT from the same related donors, achieve stable allograft function, full donor engraftment, and successful immunosuppression withdrawal through immune tolerance induction.</p><p><strong>Conclusions: </strong>Dual haplo-HCT and living donor liver transplantation (LDLT) is feasible in eligible SCD patients with end-stage liver disease.</p>","PeriodicalId":72646,"journal":{"name":"Communications medicine","volume":"5 1","pages":"354"},"PeriodicalIF":5.4000,"publicationDate":"2025-08-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12356966/pdf/","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Communications medicine","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.1038/s43856-025-01040-0","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"MEDICINE, RESEARCH & EXPERIMENTAL","Score":null,"Total":0}
引用次数: 0
Abstract
Background: Sickle cell disease (SCD) is debilitating, with age-dependent complications such as stroke and liver disease, leading to significant morbidity and early mortality in young adults. Hematopoietic stem cell transplantation (HCT) is curative treatment for SCD, but transplantation-related risks often deter its use, especially in patients with severe comorbidities. A subset of severe SCD patients with significant end-organ dysfunction may benefit from a combined approach of HCT and solid organ transplantation (SOT).
Methods: To the best of our knowledge, this report presents, for the first time, the utilization of dual orthotopic liver transplantation and haploidentical HCT (haplo-HCT) for severe SCD with advanced liver cirrhosis. Employing nonmyeloablative conditioning regimen and post-transplantation cyclophosphamide (PTCy).
Results: Both patients undergo orthotopic liver transplantation followed by haplo-HCT from the same related donors, achieve stable allograft function, full donor engraftment, and successful immunosuppression withdrawal through immune tolerance induction.
Conclusions: Dual haplo-HCT and living donor liver transplantation (LDLT) is feasible in eligible SCD patients with end-stage liver disease.
求助内容：
应助结果提醒方式：
京公网安备 11010802042870号
