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Keyword
- ADCC1
- Allogeneic hematopoietic stem cell transplant1
- Antibody dependent cellular cytotoxicity1
- CBT1
- Chronic GvHD Relapse Free Survival1
- CLPs1
- Common lymphoid progenitors1
- CRFS1
- DFS1
- Disease Free Survival1
- Gene therapy1
- Graft versus Host Disease1
- GvHD1
- HCT1
- Hematopoietic progenitor cells1
- Hematopoietic Stem Cell Transplant1
- HPCSs1
- Immune Reconstitution1
- IR1
- Non-relapse mortality1
- NRM1
- OS1
- Overall Survival1
- Post-Transplant Cyclophosphamide1
- PTCy1
ISCT Publications
2 Results
- ISCT Committee Paper
An ISCT Stem Cell Engineering Committee Position Statement on Immune Reconstitution: the importance of predictable and modifiable milestones of immune reconstitution to transplant outcomes
CytotherapyVol. 24Issue 4p385–392Published in issue: April, 2022- Alice Bertaina
- Allistair Abraham
- Carmem Bonfim
- Sandra Cohen
- Duncan Purtill
- Annalisa Ruggeri
- and others
Cited in Scopus: 2Allogeneic stem cell transplantation is a potentially curative therapy for some malignant and non-malignant disease. There have been substantial advances since the approaches first introduced in the 1970s, and the development of approaches to transplant with HLA incompatible or alternative donors has improved access to transplant for those without a fully matched donor. However, success is still limited by morbidity and mortality from toxicity and imperfect disease control. Here we review our emerging understanding of how reconstitution of effective immunity after allogeneic transplant can protect from these events and improve outcomes. - Review
Curative therapy for hemoglobinopathies: an International Society for Cell & Gene Therapy Stem Cell Engineering Committee review comparing outcomes, accessibility and cost of ex vivo stem cell gene therapy versus allogeneic hematopoietic stem cell transplantation
CytotherapyVol. 24Issue 3p249–261Published online: December 6, 2021- Alexis Leonard
- Alice Bertaina
- Carmem Bonfim
- Sandra Cohen
- Susan Prockop
- Duncan Purtill
- and others
Cited in Scopus: 5Thalassemia and sickle cell disease (SCD) are the most common monogenic diseases in the world and represent a growing global health burden. Management is limited by a paucity of disease-modifying therapies; however, allogeneic hematopoietic stem cell transplantation (HSCT) and autologous HSCT after genetic modification offer patients a curative option. Allogeneic HSCT is limited by donor selection, morbidity and mortality from transplant conditioning, graft-versus-host disease and graft rejection, whereas significant concerns regarding long-term safety, efficacy and cost limit the broad applicability of gene therapy.
