Butensky Ellen, Pakbaz Zahra, Foote Drucilla, Walters Mark C, Vichinsky Elliott P, Harmatz Paul
Department of Gastroenterology and Nutrition, Children's Hospital & Research Center at Oakland, 747 52nd St., Oakland, CA 94609, USA.
Ann N Y Acad Sci. 2005;1054:290-9. doi: 10.1196/annals.1345.038.
Treatment of hepatitis C virus (HCV) in the general population has improved over the last decade. Patients treated with peginterferon alfa (PegIFN) and ribavirin (RBV) combination therapy demonstrate overall 50-55% sustained viral response (SVR) with rates as high as 80% in patients with genotypes 2 and 3. Because RBV induces hemolysis and subsequently increases blood transfusion requirements, combination therapy has been considered contraindicated for hemoglobinopathies. This report reviews the response to interferon alfa and RBV (IFN/RBV) and PegIFN/RBV combination therapies in patients treated in the Northern California Comprehensive Thalassemia Center. A total of six thalassemia major patients were treated with IFN/RBV (n = 5; age: 4-38 years) or with PegIFN/RBV (n = 1; age: 26 years). Quantitative HCV RNA polymerase chain reaction and liver iron level assessment were completed. Transfusion volumes were obtained from patients' medical records. On IFN/RBV combination, four of five patients demonstrated SVR. The one patient on PegIFN/RBV showed end-treatment viral response after 6 months of therapy (genotype 3), but subsequently relapsed. Liver iron pretreatment level ranged from 0.2 to 22 mg/g dry weight, with a mean +/- SD of 7.9 +/- 7.7. Transfusion requirement increased by a median of 43.5% (range: 32-137%). Five of the six patients had liver iron measurements within 1 year following completion of treatment, with quantitative liver iron increasing in two patients by 2.5 mg/g dry weight, decreasing in two patients by 3 and 14 mg/g dry weight, and remaining unchanged in one patient. All patients were able to complete combination therapy, although dose reductions were required. Patients with thalassemia and high iron overload can obtain SVR after combination therapy with rates similar to those in the general population and without significant complications. Although transfusion requirements increased in most patients, iron burden was not necessarily increased.
在过去十年中,普通人群丙型肝炎病毒(HCV)的治疗情况有所改善。接受聚乙二醇干扰素α(PegIFN)和利巴韦林(RBV)联合治疗的患者总体持续病毒学应答(SVR)率为50%-55%,基因2型和3型患者的应答率高达80%。由于RBV会诱发溶血并随后增加输血需求,联合治疗一直被认为不适用于血红蛋白病患者。本报告回顾了在北加利福尼亚综合地中海贫血中心接受治疗的患者对干扰素α和RBV(IFN/RBV)以及PegIFN/RBV联合治疗的反应。共有6例重型地中海贫血患者接受了IFN/RBV(n = 5;年龄:4-38岁)或PegIFN/RBV(n = 1;年龄:26岁)治疗。完成了HCV RNA定量聚合酶链反应和肝脏铁水平评估。从患者病历中获取输血量。接受IFN/RBV联合治疗的5例患者中有4例实现了SVR。接受PegIFN/RBV治疗的1例患者在治疗6个月后显示出治疗结束时的病毒学应答(基因3型),但随后复发。治疗前肝脏铁水平范围为0.2至22mg/g干重,平均±标准差为7.9±7.7。输血需求中位数增加了43.5%(范围:32%-137%)。6例患者中有5例在治疗结束后1年内进行了肝脏铁测量,其中2例患者的定量肝脏铁增加了2.5mg/g干重,2例患者分别减少了3mg/g干重和14mg/g干重,1例患者保持不变。所有患者均能够完成联合治疗,尽管需要减少剂量。地中海贫血和高铁过载患者在联合治疗后可获得与普通人群相似的SVR率,且无明显并发症。虽然大多数患者的输血需求增加,但铁负荷不一定增加。