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Haplo-stem cell transplant post liver transplantation to cure sickle cell disease with related liver dysfunction: a
Ali D Alahmari1, Saad Alghamdi2, Reem Alasbali3
1Hematology, Stem cell transplant & Cellular therapy department, Cancer Centre of Excellence, King Faisal Specialist Hospital and Research Centre, Riyadh, 11211, Saudi Arabia. aalahmari7@kfshrc.edu.sa.
Insights
Dual haploidentical hematopoietic stem cell transplantation (haplo-HCT) combined with living donor liver transplantation (LDLT) offers a feasible curative approach for severe sickle cell disease (SCD) patients with end-stage liver disease.
Area of Science:
- Hematology
- Transplantation immunology
- Hepatology
Background:
- Sickle cell disease (SCD) causes severe complications like stroke and liver disease, leading to early mortality.
- Hematopoietic stem cell transplantation (HCT) is curative but risky, especially for patients with comorbidities.
- Combined HCT and solid organ transplantation (SOT) may benefit severe SCD patients with organ dysfunction.
Purpose of the Study:
- To report the first use of dual orthotopic liver transplantation and haploidentical HCT (haplo-HCT) in severe SCD patients with advanced liver cirrhosis.
- To evaluate the feasibility and outcomes of this combined approach.
Main Methods:
- Utilized dual orthotopic liver transplantation followed by haplo-HCT from related donors.
- Employed a nonmyeloablative conditioning regimen and post-transplantation cyclophosphamide (PTCy).
Main Results:
- Both patients achieved stable liver allograft function and full donor engraftment after haplo-HCT.
- Successful immunosuppression withdrawal was achieved through immune tolerance induction in both cases.
Conclusions:
- Dual haplo-HCT and living donor liver transplantation (LDLT) is a feasible treatment option for eligible SCD patients with end-stage liver disease.
- This combined approach demonstrates potential for curative treatment in complex SCD cases.
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.
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