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Abstract

Objectives: The only curative treatment for many patients with primary immunodeficiency disease is hematopoietic stem cell transplant. In this study, we report the transplant outcomes of patients with primary immunodeficiency diseases. Materials and Methods: Herein, we present the transplant outcomes of 20 patients with primary immunodeficiency disease seen at our center in Kayseri, Turkey, from 2010 to 2015. Results: The disease distribution of th 3 e 20 patients were as follows: 6 patients with severe combined immunodeficiency, 4 patients with hemophagocytic lymphohistiocytosis, 2 patients with chronic granulomatous disease, 2 patients with type 2 Griscelli syndrome, 2 patients with B-cell deficiency plus bone marrow failure, 1 patient with severe congenital neutropenia, 1 patient with X-linked lymphoproliferative disease, 1 patient with T-cell deficiency plus relapsed non-Hodgkin lymphoma, and 1 patient with type 1 leukocyte adhesion deficiency. Of the 20 patients, 11 received related HLA-matched, 6 received haploidentical, 2 received unrelated HLA-matched, and 1 received HLA-mismatched transplant. The median age at transplant was 21 months, and median follow-up was 5 months. Overall survival rate was 65%. Mean engraftment times for neutrophils and platelets were 14.25 ± 3.08 and 24.7 ± 11.4 days. Graft-versushost disease was observed in 30% of patients. Conclusions: Patients with primary immunodeficiency disease treated at our center had acceptable transplant outcomes. This study supports the use of hematopoietic stem cell transplant in patients with primary immunodeficiency disease.

Multicenter Outcome of Hematopoietic Stem Cell Transplantation for Primary Immune Deficiency Disorders in India

“…Similar results have been seen in the EBMT (16)(17)(18) and Latin American groups (19) with data from focused groups showing improvement in overall survival. Significant challenges in early diagnosis, supportive care and financial constraints need to be overcome in low and middle income countries as reported by groups from Brazil, Jordan, China, Iran and Turkey (20)(21)(22)(23)(24)(25)(26).…”

First report of an SH2D1A mutation associated with X-linked lymphoproliferative disease in Turkey

“…Death is generally attributable to liver failure with hepatic encephalopathy or bone marrow failure with fatal hemorrhages in various organs [ 5 ]. The only curative treatment of XLP is hematopoietic stem cell transplantation [ 6 ].…”