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Hematopoietic stem cell transplantation from unrelated donors in children with DOCK 8 deficiency
Author(s) -
Uygun Dilara Fatma K.,
Uygun Vedat,
Reisli İsmail,
Keleş Sevgi,
Özen Ahmet,
Yılmaz Mustafa,
Sayar Esra H.,
Daloğlu Hayriye,
Öztürkmen Seda I.,
Çakı Suar,
Karasu Gülsün T.,
Yeşilipek Akif
Publication year - 2017
Publication title -
pediatric transplantation
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.457
H-Index - 69
eISSN - 1399-3046
pISSN - 1397-3142
DOI - 10.1111/petr.13015
Subject(s) - medicine , dock , transplantation , hematopoietic stem cell transplantation , stem cell , immunology , hematopoietic cell , haematopoiesis , microbiology and biotechnology , genetics , biology
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.