1. Allogeneic stem cell transplant in primary hemophagocytic lymphohistiocytosis - a single-center experience.
- Author
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Hussain F, Hussain M, Kerio AA, Ghafoor T, Khattak TA, Chaudhry QUN, Shahbaz N, Ali Khan M, and Iftikhar R
- Subjects
- Humans, Male, Female, Retrospective Studies, Adolescent, Child, Child, Preschool, Adult, Infant, Transplantation Conditioning methods, Transplantation, Homologous, Allografts, Young Adult, Piebaldism therapy, Middle Aged, Survival Rate, Disease-Free Survival, Follow-Up Studies, Primary Immunodeficiency Diseases, Perforin, Lymphohistiocytosis, Hemophagocytic therapy, Lymphohistiocytosis, Hemophagocytic mortality, Hematopoietic Stem Cell Transplantation
- Abstract
Hemophagocytic Lymphohistiocytosis (HLH) is a rare disorder of immune dysregulation characterized by fever, cytopenias, and splenomegaly. Its primary form poses a therapeutic challenge due to its high fatality when left untreated. We retrospectively analyzed 28 patients who underwent related-donor allogeneic stem cell transplant for primary HLH from 2010 to 2021. Among them were 10 cases of familial HLH, 8 cases of Griscelli syndrome type 2, and 1 case each with PRF1 and STX11 mutations. All the patients underwent transplants with reduced-intensity or myeloablative conditioning and 26 of them achieved neutrophil engraftment at a median of day + 14. The donors were either fully matched (68%) or haploidentical (32%). With a median follow-up of 1 year, overall survival was 68% (n = 19) and disease-free survival was 64.4% (n = 18). OS was better in patients transplanted with a sibling donor (compared to parent donor), who achieved complete donor chimerism, and those transplanted early in the course of the disease (diagnosis to transplant duration less than 6 months)., (© 2024. The Author(s), under exclusive licence to Springer-Verlag GmbH Germany, part of Springer Nature.)
- Published
- 2024
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