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

被引：12

作者：

Uygun, Dilara Fatma K. ^{[1
]}

Uygun, Vedat ^{[2
]}

Reisli, Ismail ^{[3
]}

Keles, Sevgi ^{[3
]}

Ozen, Ahmet ^{[4
]}

Yilmaz, Mustafa ^{[5
]}

Sayar, Esra H. ^{[3
]}

Daloglu, Hayriye ^{[2
]}

Ozturkmen, Seda I. ^{[2
]}

Caki, Suar ^{[6
]}

Karasu, Gulsun T. ^{[6
]}

Yesilipek, Akif ^{[2
]}

机构：

[1] Antalya Training & Res Hosp, Dept Pediat Immunol, Antalya, Turkey

[2] Bahcesehir Univ, MedicalPk Antalya Hosp, Bone Marrow Transplantat Unit, Fac Med,Dept Pediat, Antalya, Turkey

[3] Necmettin Erbakan Univ, Div Pediat Immunol & Allergy, Meram Med Fac, Konya, Turkey

[4] Marmara Univ, Div Pediat Allergy & Immunol, Fac Med, Istanbul, Turkey

[5] Cukurova Univ, Div Pediat Allergy & Immunol, Fac Med, Adana, Turkey

[6] Bahcesehir Univ, MedicalPk Goztepe Hosp, Bone Marrow Transplantat Unit, Dept Pediat,Fac Med, Istanbul, Turkey

来源：

PEDIATRIC TRANSPLANTATION | 2017年 / 21卷 / 07期

关键词：

DOCK8; deficiency; HSCT; hyperimmunoglobulin E syndrome; unrelated donor; CYTOKINESIS; 8; DEFICIENCY; VERSUS-HOST-DISEASE; GRAFT; CYCLOPHOSPHAMIDE; DEDICATOR; TOXICITY;

D O I：

10.1111/petr.13015

中图分类号：

R72 [儿科学];

学科分类号：

100202 ;

摘要：

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.

引用

页数：4

共 20 条

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