Case report: HLA-haploidentical hematopoietic cell transplant with posttransplant cyclophosphamide in a patient with leukocyte adhesion deficiency type I

被引：3

作者：

Yamashita, Motoi ^{[1
]}

Eguchi, Shiori ^{[1
]}

Tomomasa, Dan ^{[1
]}

Kamiya, Takahiro ^{[2
]}

Niizato, Daiki ^{[1
]}

Mitsuiki, Noriko ^{[1
]}

Isoda, Takeshi ^{[1
]}

Funakoshi, Hanako ^{[3
]}

Mizuno, Yuki ^{[4
]}

Okamoto, Kentaro ^{[4
]}

Nguyen, Tuan Minh ^{[5
]}

Takada, Hidetoshi ^{[6
]}

Takagi, Masatoshi ^{[1
]}

Imai, Kohsuke ^{[7
,8
]}

Morio, Tomohiro ^{[1
]}

Kanegane, Hirokazu ^{[9
]}

机构：

[1] Tokyo Med & Dent Univ, Grad Sch Med & Dent Sci, Dept Pediat & Dev Biol, Tokyo, Japan

[2] Tokyo Med & Dent Univ, Clin Res Ctr, Div Clin Management, Tokyo, Japan

[3] Tokyo Metropolitan Childrens Med Ctr, Dept Pediat, Div Infect Dis, Tokyo, Japan

[4] Tokyo Med & Dent Univ Hosp, Dept Pediat Surg, Tokyo, Japan

[5] Childrens Hosp 1, Dept Hematol, HCM, Ho Chi Minh City, Vietnam

[6] Univ Tsukuba, Fac Med, Dept Child Hlth, Tsukuba, Japan

[7] Tokyo Med & Dent Univ, Dept Community Pediat Perinatal & Maternal Med, Tokyo, Japan

[8] Natl Def Med Coll, Dept Pediat, Tokorozawa, Japan

[9] Tokyo Med & Dent Univ, Grad Sch Med & Dent Sci, Dept Child Hlth & Dev, Tokyo, Japan

来源：

FRONTIERS IN IMMUNOLOGY | 2022年 / 13卷

关键词：

leukocyte adhesion deficiency; posttransplant cyclophosphamide; hematopoietic cell transplantation; graft-versus-host disease; inborn errors of immunity; RECONSTITUTION;

D O I：

10.3389/fimmu.2022.1020362

中图分类号：

R392 [医学免疫学]; Q939.91 [免疫学];

学科分类号：

100102 ;

摘要：

Leukocyte adhesion deficiency type I (LAD-I) is a rare autosomal recessive inborn error of immunity (IEI) caused by the defects in CD18, encoded by the ITGB2 gene. LAD-I is characterized by defective leukocyte adhesion to the vascular endothelium and impaired migration of leukocytes. Allogeneic hematopoietic cell transplant (HCT) is the only curative treatment for LAD-I. In an absence of ideal donor for HCT, human leukocyte antigen (HLA)-haploidentical HCT is performed. Posttransplant cyclophosphamide (PT-CY) is a relatively new graft-versus-host disease (GVHD) prophylactic measure and has been increasingly used in HLA-haploidentical HCT for malignant and nonmalignant diseases. However, experience in using PT-CY for rare IEIs, such as LAD-I, is very limited. We report a case of LAD-I successfully treated with HLA-haploidentical HCT with PT-CY. Complete chimerism was achieved, and the patient was cured. Her transplant course was complicated by mild GVHD, cytomegalovirus reactivation and veno-occlusive disease/sinusoidal obstruction syndrome, which were successfully treated. HLA-haploidentical HCT with PT-CY is a safe and effective option for patients with LAD-I when HLA-matched donors are unavailable.

引用

页数：7

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