Donato H, Kohan R, Picón A, Rovó A, Rapetti M C, Schvartzman G, Lavergne M, de Galvagni A, Rosso A, Rendo P
Clinical Research Area, Bio Sidus S.A. Laboratory, Buenos Aires, Argentina.
J Pediatr Hematol Oncol. 2001 Dec;23(9):598-603. doi: 10.1097/00043426-200112000-00009.
To investigate alpha-interferon (IFN) therapy for children with chronic idiopathic thrombocytopenic purpura (ITP).
Patients with refractory ITP lasting more than 12 months from diagnosis were included if they had platelet counts <50 x 10(9)/L and had received no treatment during the past month. Patients received IFN (3 x 10(6) U/m2 per dose), three times per week for 4 weeks; if partial (<150 x 10(9)/L) or no response was obtained, the same dose was continued for another 8 weeks. In patients with favorable response and subsequent decrease to pre-treatment values, an additional 4 weeks of treatment could be administered.
Fourteen patients (ages 4-20 y) receiving 17 IFN courses were included. Mean initial platelet count was 29 +/- 15 x 10(9)/L. A significant increase was achieved during 14 of 17 courses (82.4%). All but two responses were transitory, and platelets returned to initial values after IFN discontinuation (mean 44 +/- 26 days). Considering the best response achieved by each patient, we observed: 1) 10 patients who achieved a sustained improvement of platelet count throughout the treatment period, decreasing to initial values after therapy was stopped; 2) one patient who achieved platelet count >150 x 10(9)/L, remaining with normal platelets at 18 months; 3) one patient who achieved platelet count >150 x 10(9)/L, remaining with platelets between 100 and 140 x 10(9)/L at 48 months; 4) one patient who had no response; and 5) one patient in whom therapy worsened the thrombocytopenia. A mild to moderate flu-like syndrome and a moderate decrease of the absolute neutrophil count were the only side effects observed.
Interferon therapy induces a significant increase of platelet count and seems to be a valid alternative therapy to attempt the achievement of prolonged remission in refractory ITP, to defer splenectomy in younger children, or to improve platelet count before planned splenectomy.
探讨α-干扰素(IFN)治疗儿童慢性特发性血小板减少性紫癜(ITP)的效果。
纳入诊断后难治性ITP持续超过12个月的患者,这些患者血小板计数<50×10⁹/L且在过去1个月内未接受治疗。患者接受IFN(每剂量3×10⁶U/m²),每周3次,共4周;若部分缓解(<150×10⁹/L)或无反应,则继续给予相同剂量治疗8周。对反应良好且随后血小板计数降至治疗前水平的患者,可再给予4周治疗。
14例患者(年龄4 - 20岁)接受了17个IFN疗程的治疗。初始血小板计数平均值为29±15×10⁹/L。17个疗程中有14个(82.4%)血小板计数显著升高。除2例反应外,所有反应均为短暂性,IFN停用后血小板计数恢复至初始值(平均44±26天)。考虑每位患者的最佳反应,我们观察到:1)10例患者在整个治疗期间血小板计数持续改善,治疗停止后降至初始值;2)1例患者血小板计数>150×10⁹/L,18个月时血小板维持正常;3)1例患者血小板计数>150×10⁹/L,48个月时血小板计数维持在100 - 140×10⁹/L之间;4)1例患者无反应;5)1例患者治疗后血小板减少症加重。观察到的唯一副作用是轻度至中度的流感样综合征和绝对中性粒细胞计数中度下降。
干扰素治疗可使血小板计数显著升高,似乎是难治性ITP实现长期缓解、推迟年幼儿童脾切除术或在计划脾切除术前改善血小板计数的一种有效替代疗法。