PHASE II EFFICACY STUDY OF ROFERON A (R022-8181/002) 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，大多数患者对 干扰素，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。似乎有非常多的 很少有患者在接受治疗后不需要进一步治疗 持续12或18个月的干扰素治疗。正因为如此 结果，我们选择对以下患者持续给予干扰素 最初对这种药物有反应。在53名可评估的患者中( 参加这项研究的56人)，有一人完全缓解，41人 部分缓解，1例轻微缓解，9例病情稳定， 仅1例患者病情进展。14名患者继续接受治疗 无中断接受干扰素治疗，持续时间中位数为 连续使用干扰素治疗9.2年。34名患者 停用干扰素的原因有很多，最常见的是 获得性干扰素耐药性的发生与 干扰素抗体。对干扰素的耐药性很早就表现出来了， 在治疗的前18个月，除两例外。一个重要的 在这项研究中发现的是持续缓慢但意义重大的血液学 18个月后粒细胞和血小板绝对值的改善 从而表明延长治疗时间会导致 持续受益，而不是产生抗体 干扰素耐药性的发展。尽管从这项研究可以清楚地看到 毛细胞性白血病是可以长期控制的 干扰素，需要更长时间的随访才能确定是否持续 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, 41 partial remissions, 1 minor response, 9 patients with stable disease and only 1 patient with disease progression. Fourteen patients continue to receive interferon without interruption with a median duration of continuous interferon treatment of 9.2 years. Thirty-four patients discontinue 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 manifest early, in the first 18 months of treatment, except in two cases. 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.