| Autor: |
Ambreen Pandrowala, MBBS, DNB (Paediatrics), Ajay Narayan Sharma, MBBS, DNB (Paediatrics), Manasa Kakunje, MBBS, DNB (Paediatrics), Minnie Bodhanwala, BDS, MBA, Prashant Hiwarkar, MD, FRCPath, PhD |
| Jazyk: |
angličtina |
| Rok vydání: |
2023 |
| Předmět: |
|
| Zdroj: |
Journal of Allergy and Clinical Immunology: Global, Vol 2, Iss 3, Pp 100106- (2023) |
| Druh dokumentu: |
article |
| ISSN: |
2772-8293 |
| DOI: |
10.1016/j.jacig.2023.100106 |
| Popis: |
Background: Biallelic mutations in the dedicator of cytokinesis 8 (DOCK8) gene were identified as the cause of combined immunodeficiency in 2009. Survival rates without hematopoietic stem cell transplant in patients with DOCK8 deficiency decline from 87% at 10 years to 33% at 30 years. Hematopoietic stem cell transplant is therefore the recommended treatment for cure of DOCK8 deficiency. However, patients with DOCK8 deficiency have multiple infectious comorbidities; hence, they cannot tolerate myeloablative conditioning. Reduced intensity conditioning reduces the risk of transplant-related mortality but increases the possibility of mixed chimerism. Mixed chimerism in children with immunodeficiency increases the risk of autoimmunity and the need for long-term immunoglobulin infusion. Objective: Here we have sought to devise a strategy for reducing the possibility of mixed chimerism without increasing the risk of transplant-related mortality. Methods: To balance the risk of transplant-related mortality and mixed chimerism, we used treosulfan-based reduced toxicity conditioning with a high CD34+ cell dose and differential T-cell capping for HLA-matched and haploidentical transplants. Results: We are able to report that by using the aforementioned novel strategy, we achieved excellent transplant outcomes in the first cohort of high-risk patients with DOCK8 deficiency from India. Conclusion: High CD34+ cell dose and reduced toxicity conditioning can achieve full donor chimerism in DOCK8 deficiency. |
| Databáze: |
Directory of Open Access Journals |
| Externí odkaz: |
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