Successful haploidentical bone marrow transplantation in Griscelli syndrome type 2 with non-busulfan-based regimen and post-transplantation cyclophosphamide: a case report and review of the literature.

Autor: Yamada, Saori, Maruyama, Yuta, Saito, Shoji, Komori, Kazutoshi, Morokawa, Hirokazu, Okura, Eri, Hirabayashi, Koichi, Furui, Yu, Kurata, Takashi, Nishioka, Makoto, Fukuyama, Tetsuhiro, Sakashita, Kazuo, Nakazawa, Yozo
Předmět:
Zdroj: Pediatric Hematology & Oncology; 2024, Vol. 41 Issue 4, p296-300, 5p
Abstrakt: This article discusses a case report of a successful bone marrow transplantation in a patient with Griscelli syndrome type 2 (GS2), a rare genetic disorder characterized by silver-gray hair and hypopigmentation. The patient underwent transplantation using a non-busulfan-based regimen and post-transplantation cyclophosphamide administration, resulting in a successful outcome with minimal adverse events. The article also reviews the literature on GS2 patients who received transplantation with non-busulfan-based regimens, highlighting the lower incidence of veno-occlusive disease in this group compared to those who received busulfan-based regimens. The study suggests that avoiding busulfan-based regimens may improve outcomes for transplantation in GS2 patients. [Extracted from the article]
Databáze: Complementary Index