Hematopoietic stem cell transplantation from unrelated donors in children with DOCK8 deficiency.

Abstract:

:DIDS is a unique form of combined immune deficiency characterized by an unusual susceptibility to cutaneous viral infections, severe allergies with eosinophilia and elevated immunoglobulin E titers, autoimmunity, and cancer. HSCT is considered the standard of care for this deadly disease. We have retrospectively analyzed the outcome of allogeneic HSCT from unrelated donors in patients with DIDS. Data from four patients, with five transplants, are presented. All patients received transplants from unrelated donors' BM, except for one patient who received a cord blood transplant. The conditioning regimens were based on myeloablative protocols for BM derived transplants; a NM regimen was pursued for the patient who received a cord blood transplant, which resulted in graft rejection. Although recurrent pneumonia and skin infections resolved immediately after transplantation, all patients subsequently developed human herpesvirus infection, including cutaneous herpetic lesions, cytomegalovirus reactivation, and zona zoster, which could be attributed to the use of ATG. Despite the presence of serious morbidities prior to transplantation, all patients recovered successfully. DIDS can be successfully treated with allogeneic HSCT from unrelated donors following a myeloablative conditioning regimen, with a reasonable safety profile.

journal_name

Pediatr Transplant

authors

Uygun DFK,Uygun V,Reisli İ,Keleş S,Özen A,Yılmaz M,Sayar EH,Daloğlu H,Öztürkmen SI,Çakı S,Karasu GT,Yeşilipek A

doi

10.1111/petr.13015

subject

Has Abstract

pub_date

2017-11-01 00:00:00

issue

7

eissn

1397-3142

issn

1399-3046

journal_volume

21

pub_type

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