The role of HLA matching in unrelated donor hematopoietic stem cell transplantation for sickle cell disease in Europe

被引：13

作者：

Gluckman, Eliane ^{[1
,2
]}

de la Fuente, Josu ^{[3
]}

Cappelli, Barbara ^{[1
,2
]}

Scigliuolo, Graziana M. ^{[1
,2
]}

Volt, Fernanda ^{[1
,2
]}

Tozatto-Maio, Karina ^{[1
,2
,4
]}

Rocha, Vanderson ^{[1
,2
,5
]}

Tommaso, Mina ^{[6
]}

O'Boyle, Farah ^{[3
]}

Smiers, Frans ^{[7
]}

Da Cunha-Riehm, Claudia Bettoni ^{[8
]}

Calore, Elisabetta ^{[9
]}

Bonanomi, Sonia ^{[10
]}

Graphakos, Stelios ^{[11
]}

Paisiou, Anna ^{[11
]}

Albert, Michael H. ^{[12
]}

Ruggeri, Annalisa ^{[12
,13
,14
]}

Zecca, Marco ^{[6
]}

Lankester, Arjan C. ^{[7
]}

Corbacioglu, Selim ^{[15
]}

机构：

[1] Univ Paris, Hop St Louis, Eurocord, Paris, France

[2] Ctr Sci Monaco, Monacord, Monaco, Monaco

[3] St Marys Hosp, Dept Paediat, London, England

[4] Univ Sao Paulo, Fac Med Ribeirao Preto, Ribeirao Preto, Brazil

[5] Hosp Sirio Libanes, Bone Marrow Transplant Unit, Sao Paulo, Brazil

[6] Fdn IRCCS Policlin San Matteo, Pediat Hematol Oncol, Pavia, Italy

[7] Leiden Univ, Med Ctr, Dept Pediat, Leiden, Netherlands

[8] Med Sch Hannover, Dept Pediat Hematol & Oncol, Hannover, Germany

[9] Azienda Osped Univ Padova, Dept Womens & Childrens Hlth, Pediat Hematol Oncol Unit, Padua, Italy

[10] Univ Milano Bicocca, MBBM Fdn, San Gerardo Hosp, Pediat Clin Hematooncol Dept, Monza, Italy

[11] Aghia SophiaChildrens Hosp, Stem Cell Transplant Unit, Athens, Greece

[12] Dr von Haunersches Univ Childrens Hosp, Pediat Hematol & Oncol, Munich, Germany

[13] IRCCS Osped Pediatr Bambino Gesu, Dept Pediat Hematol & Oncol, Rome, Italy

[14] Cellular Therapy & Immunobiol WP EBMT, Leiden, Netherlands

[15] Univ Regensburg, Dept Pediat Hematol Oncol & Stem Cell Transplanta, Regensburg, Germany

来源：

BONE MARROW TRANSPLANTATION | 2020年 / 55卷 / 10期

关键词：

BONE-MARROW-TRANSPLANTATION; CORD BLOOD TRANSPLANTATION; RELAPSE-FREE SURVIVAL; QUALITY-OF-LIFE; CHILDREN; CHIMERISM; PATIENT; GRAFTS; CURE;

D O I：

10.1038/s41409-020-0847-z

中图分类号：

Q6 [生物物理学];

学科分类号：

071011 ;

摘要：

We report the results of an analysis of unrelated allogeneic hematopoietic stem cell transplantations (HSCT) in 71 patients with sickle cell disease (SCD) transplanted in EBMT centers between 2005 and 2017. Median age was 9.3 years; graft type was bone marrow in 79% and peripheral blood in 21%. Recipient-donor HLA match at high resolution typing was 10/10 in 31, 9/10 in 20, and 8/10 in 4 patients; the other patients had intermediate resolution typing. The most frequent conditioning regimens were fludarabine-thiotepa-treosulfan (64%) or busulfan-cyclophosphamide (12%). Cumulative incidence of neutrophil engraftment was 92%; platelet engraftment was 90%. Eleven patients (15%) experienced graft failure. Grade II-IV acute graft-vs.-host disease (GvHD) was 23%; 3-year chronic GvHD was 23%. Three-year overall survival (OS) was 88 +/- 4%. GRFS was 62 +/- 6%. HLA matching was the most significant risk factor for OS: 3-year OS was 96 +/- 4% in 10/10 group vs. 75 +/- 10% in 9-8/10 (p = 0.042); GRFS was 69 +/- 9% vs. 50 +/- 12% (p = 0.114), respectively. In conclusion, unrelated donor HSCT is a valid option for SCD patients who lack an HLA-identical sibling donor, preferably in the context of clinical trials. Using a 10/10 HLA-matched unrelated donor yields better survival indicating that HLA matching is an important donor selection factor in this nonmalignant disease.

引用

页码：1946 / 1954

页数：9

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