PHASE II EFFICACY STUDY OF ROFERON A (RO22-8181/002) IN HAIRY CELL LEUKEMIA

这项研究始于干扰素α-2a对患者的疗效研究。 患有毛细胞白血病。据观察，大多数患者都有反应 对干扰素的反应，但获得的完全应答很少。 在其他地方进行的研究证实了低的完全缓解率。 一旦干扰素停止，几乎一致的疾病进展 观察需要重新治疗的情况。似乎有一些 极少数(如果有)患者在治疗后不再需要进一步治疗 接受12或18个月的持续干扰素治疗。因为 在这些发现中，我们选择持续给予干扰素来 最初对这种药物有反应的患者。在53个可评估的 患者(参加这项研究的56名患者中)，有一名患者 缓解，40例部分缓解，2例轻微反应，9例 病情稳定，仅1例病情进展。在最后一次 去年，其中一名有轻微反应的患者现在获得了 部分缓解。32名患者继续接受干扰素治疗 无中断，持续时间中位数为干扰素 治疗时间为6.5年。21例患者停用干扰素治疗 原因是多方面的，最常见的是后天的发展 干扰素抵抗与干扰素抗体相关。这个 对干扰素的耐药性很早就表现出来，在前18个月 治疗。这项研究中的一个重要发现是持续缓慢的， 但显著的，血液学改善绝对粒细胞和 治疗18个月以上的血小板计数，从而表明 延长治疗会带来持续的益处，而不是 抗体的产生与随后的干扰素的发展 抵抗。尽管从这项研究中可以清楚地看到毛细胞 白血病可以用干扰素长期控制，时间更长 有必要进行随访，以确定是否需要继续治疗 干扰素治疗优于间歇治疗。

This study began as an efficacy study of interferon Alpha-2a in patients with hairy cell leukemia. It was observed that most patients responded to interferon, but that very few complete responses were being obtained. Studies being done elsewhere confirmed the low complete remission rate. Once interferon was stopped, nearly uniformly disease progression requiring reinstitution of therapy was observed. There appear to be very few if any patients who will not require further therapy after receiving 12 or 18 months of continuous interferon treatment. Because of these findings, we opted to administer interferon continuously to patients who were initially responsive to this drug. of the 53 evaluable patients (of the 56 entered on this study), there was one complete remission, 40 partial remissions, 2 minor responses, 9 patients with stable disease and only 1 patient with disease progression. In the last year, one of the patients with a minor response has now obtained a partial remission. Thirty-two patients continue to receive interferon without interruption with a median duration of continuous interferon treatment of 6.5 years. Twenty-one patients discontinued interferon for a variety of reasons, the most common being the development of acquired interferon resistance in association with interferon antibodies. The resistance to interferon was manifested early, in the first 18 months of treatment. An important finding in this study is the continued slow, but significant, hematologic improvement in absolute granulocyte and platelet counts beyond 18 months of therapy, thereby indicating that prolonged treatment results in continued benefit rather than the production of antibodies with subsequent development of interferon resistance. Although it is clear from this study that hairy cell leukemia can be controlled in the long-term with interferon, longer follow-up will be necessary to determine if continuous therapy with interferon is better than intermittent therapy.