Gabelli Maria, Marzollo Antonio, Notarangelo Lucia Dora, Basso Giuseppe, Putti Maria Caterina
Department of Women's and Children's Health, Pediatric Onco-Hematology Unit, University of Padova, Padova, Italy.
Pediatric Onco-Hematology and BMT Unit, Children's Hospital, Spedali Civili of Brescia, Brescia, Italy.
Pediatr Blood Cancer. 2017 Dec;64(12). doi: 10.1002/pbc.26692. Epub 2017 Jun 23.
Wiskott-Aldrich syndrome (WAS) is an inherited X-linked disorder characterized by microthrombocytopenia, immunodeficiency, and eczema. Hematopoietic stem cell transplantation (HSCT) is the treatment of choice. Eltrombopag, a thrombopoietin receptor agonist, may be useful to prevent bleeding while awaiting HSCT. We present a case of a male with WAS, profound thrombocytopenia, and bleeding diathesis successfully managed with eltrombopag before HSCT. Eltrombopag was given for 32 weeks obtaining a stable platelet count without any platelet transfusion. The patient did not experience any bleeding symptom. Eltrombopag may be a suitable therapeutic option for patients with WAS and severe thrombocytopenia as "bridge" to definitive cure.
威斯科特-奥尔德里奇综合征（WAS）是一种遗传性X连锁疾病，其特征为血小板减少、免疫缺陷和湿疹。造血干细胞移植（HSCT）是首选治疗方法。艾曲泊帕，一种血小板生成素受体激动剂，在等待HSCT期间可能有助于预防出血。我们报告一例患有WAS、严重血小板减少和出血倾向的男性患者，在HSCT前成功使用艾曲泊帕进行治疗。给予艾曲泊帕32周，血小板计数稳定，无需任何血小板输注。患者未出现任何出血症状。艾曲泊帕可能是WAS和严重血小板减少患者作为确定性治愈“桥梁”的合适治疗选择。
