| Autor: |
M, Al-Mofareh, M, Ayas, A, Al-Seraihy, K, Siddiqui, A, Al-Jefri, I, Ghemlas, H, Alsaedi, H, El-Solh, S, Al-Sweedan, B, Al-Saud, H, Al-Mousa, H, Al-Dhekri, R, Arnaout, R, Mohammed, S, Al-Muhsen, A, Al-Ahmari |
| Rok vydání: |
2019 |
| Předmět: |
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| Zdroj: |
Bone marrow transplantation. 55(10) |
| ISSN: |
1476-5365 |
| Popis: |
In 2010, we reported the outcome of hematopoietic stem cell transplantation (HSCT) in 11 children with Griscelli syndrome type 2 (GS2). We report here the update on this cohort to include 35 patients. Twenty-seven (77%) patients received conditioning regimen including busulfan, cyclophosphamide with etoposide. Eight (23%) were given busulfan, fludarabine. Thiotepa was added to busulfan and fludarabine regimen in two patients; one received haploidentical marrow and one unrelated cord blood. Posttransplant clinical events included veno-occlusive disease (n = 7), acute (n = 8), or chronic (n = 1) graft-versus-host disease II-IV. With a mortality rate of 37.1% (n = 13) and a median follow-up of 87.7 months of the survivors, 5-year cumulative probability of overall survival (OS) for our cohort of patients was 62.7% (±8.2%). Cumulative probability of 5-year OS was significantly better in those who did not have hemophagocytic lymphohistiocytosis (HLH) prior to HSCT (100% vs. 53.3 ± 9.5%, P value: 0.042). Of the 16 patients with neurologic involvement before HSCT, 8 survived and 3 presented sequelae. OS at 5-year was 50 ± 12.5% and 73.3 ± 10.2% (P value: 0.320) in patients with and without CNS involvement, respectively. In conclusion, HSCT in patients with GS2 is potentially curative with long-term disease-free survival. Early HSCT before the development of the accelerated phase is associated with a better outcome. |
| Databáze: |
OpenAIRE |
| Externí odkaz: |
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