Voxelotor treatment of a patient with sickle cell disease and very severe anemia.
Voxelotor treatment of a patient with sickle cell disease and very severe anemia.
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Voxelotor 对患有镰状细胞病和非常严重贫血的患者进行治疗。
DOI：
10.1002/ajh.25389
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发表时间：
2019
影响因子：
12.8
通讯作者：
Thein,SweeLay
中科院分区：
文献类型：
--
作者：
Shet,ArunS;Mendelsohn,Laurel;Harper,Julia;Ostrowski,David;Henry,EricR;Gwaabe,Eveline;Nichols,Jim;Alayash,AbduI;Eaton,WilliamA;Thein,SweeLay
The case involves a 38 year old female of Nigerian descent with sickle cell anemia (SCA, βS/βS genotype), no α-globin gene deletion and no glucose 6-phosphate dehydrogenase deficiency. Over the past two decades, the patient received routine care at the National Institutes of Health (NIH) participating in several studies. During childhood, the patient’s disease course was mild but after 24 years of age, she developed numerous sickle-related complications including osteomyelitis, acute chest syndrome (ACS), multi-organ system failure, chronic renal insufficiency, cholelithiasis, secondary iron overload, cardiomyopathy, and sickle hepatopathy.SCA specific therapies during this period included hydroxyurea (HU, between 2001 and 2009) with minimal clinical or hemoglobin improvement. Decitabine therapy was attempted to improve her hemoglobin but had to be discontinued largely due to thrombocytosis. A phase I trial of the anti-sickling agent AES-103 was also discontinued after 3 months due to unexplained aplastic crisis. Empiric trials of oral magnesium and omega 3 fatty acids provided no measurable clinical benefit. In February 2014, she was admitted for acute painful crisis that triggered ACS and multi-organ failure requiring hemodialysis for approximately 4 weeks and transfusion with 8 units of packed red blood cells (PRBCs, 4 with exchange transfusion and two separate simple transfusions of two units each).
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