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低资源国家(尼日利亚)首例镰状细胞病患者异体造血干细胞移植成功:病例报告

First successful allogeneic hematopoietic stem cell transplantation for a sickle cell disease patient in a low resource country (Nigeria): a case report.

作者信息

Bazuaye Nosakhare, Nwogoh Benedict, Ikponmwen Douglas, Irowa Omorodion, Okugbo Stanley, Isa Ime, Ighodaro Emmanuel, Aina Yetunde-Isreal, Osaguona Anthony, Idemudia Osagie, Iyoha Osaretin, Ighosewe Okiroro, Osaghae Dominic, Bucher Christoph

机构信息

Department of Hematology, University of Benin, Teaching Hospital, Benin, Nigeria.

Department of Surgery, University of Benin, Teaching Hospital, Benin, Nigeria.

出版信息

Ann Transplant. 2014 May 5;19:210-3. doi: 10.12659/AOT.890248.

Abstract

BACKGROUND

Sickle cell disease (SCD) has a prevalence of 2-3% in Nigeria (population: over 150 million). We present our first allogeneic hematopoietic stem cell transplantation (HSCT) for a 7-year-old patient with severe sickle cell anemia and debilitating right-sided hemi-paraparesis.

CASE REPORT

Conditioning was with (Reduced Intensity Conditioning (FLU/BU).[Fludarabine 160 mg/m2 (days -6 to -2) and Busulphan 16 mg/kg (4×25 mg 6 hly days -5 to -2) and Anti-thymocyte globulin(ATG)(ATGAM) total dose 500 mg (days -6 to -4)]. Graft versus Host Disease (GVHD) prophylaxis was with Cyclosporine A (2×50 mg daily) and Mycophenolate Mofetil (2×500 mg/day). Stem cell source was bone marrow harvested on the 28 September 2011 with 9.8×108 nucleated cells/kg in a total volume of 900 mL from his 14-year-old HLA-matched sibling (6/6). Neutrophil and platelet engraftment was day +18 and +21, respectively. At day +70 full blood count was a total white blood cell count of 3100/µl, absolute Neutrophil count 1200/µl, Hemoglobin (Hb) 11.3 g/dl, Platelet 198,000/µl, Hemoglobin phenotype AA, and no acute or chronic GVHD. He is clinically stable with a Chimerism at 2 years post-HSCT of 95% and responding to physiotherapy.

CONCLUSIONS

We have successfully performed a stem cell transplanted in a 7-year-old Sickle Cell Anemia case. With the assistance of Government and improved Health Insurance Policy, we could make HSCT available as a cure for many Nigerians with both malignant and non-malignant disorders.

摘要

背景

镰状细胞病(SCD)在尼日利亚的患病率为2%-3%(人口超过1.5亿)。我们为一名患有严重镰状细胞贫血且右侧半身轻瘫的7岁患者进行了首例异基因造血干细胞移植(HSCT)。

病例报告

预处理方案为减低剂量预处理(氟达拉滨/白消安)[氟达拉滨160mg/m²(-6至-2天),白消安16mg/kg(-5至-2天,每天4次,每次25mg,每6小时一次)以及抗胸腺细胞球蛋白(ATG)(抗胸腺细胞球蛋白)总剂量500mg(-6至-4天)]。移植物抗宿主病(GVHD)预防方案为环孢素A(每日2次,每次50mg)和霉酚酸酯(每日2次,每次500mg)。干细胞来源为2011年9月28日采集的骨髓,来自其14岁HLA配型相合的同胞(6/6),有核细胞数为9.8×10⁸个/kg,总体积为900mL。中性粒细胞和血小板植入分别在+18天和+21天。在+70天时,全血细胞计数显示白细胞总数为3100/µl,绝对中性粒细胞计数为1200/µl,血红蛋白(Hb)为11.3g/dl,血小板为198,000/µl,血红蛋白表型为AA,且无急性或慢性GVHD。他临床稳定,HSCT后2年嵌合率为95%,对物理治疗有反应。

结论

我们成功地为一名7岁镰状细胞贫血患者进行了干细胞移植。在政府的协助和完善的医疗保险政策下,我们可以使HSCT成为许多患有恶性和非恶性疾病的尼日利亚人的治疗方法。

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