Vancine-Califani Suley Mara Chaddad, De Paula Erich Vinicius, Ozelo Margareth Castro, Orsi Fernanda Loureiro Andrade, Fabri Daniela Ramos, Annichino-Bizzacchi Joyce Maria
Hematology and Hemotherapy Center, State University of Campinas, Campinas, SP, Brazil.
Platelets. 2008 Nov;19(7):489-95. doi: 10.1080/09537100802315110.
In adults with immune thrombocytopenic purpura (ITP), steroids are usually proposed as first-line therapy, but long-term complete responses are obtained in no more than 20% of patients. For the remaining patients, splenectomy is considered the treatment of choice, with reported "cure" rates from 60-70%. However, the inherent risks of surgery and sepsis after splenectomy without a guarantee of success justify the search for strategies aimed to avoid splenectomy. Here we retrospectively evaluated the results of dapsone treatment in ITP patients that failed first-line therapy with steroids. These patients received dapsone 100 mg/day for a minimum of 30 days before splenectomy was considered. Efficacy was defined as a sustained rise in platelet counts (>50 x 10(9)/l) clearly attributed to dapsone treatment. Among 52 steroid-dependent or refractory patients, dapsone resulted in sustained increases in platelet counts in 44.2% of patients, after a median follow-up of 21.10 months after treatment initiation. The long-term efficacy of dapsone in this setting is further corroborated by the observation that none of the "responding" patients required splenectomy in the follow-up, compared to 69.0% of the "non-responding" patients. Dapsone-related adverse events were mild and promptly reversed by treatment withdrawal. The results of our retrospective analysis suggest that dapsone is a safe and effective second-line agent for steroid-dependent or refractory ITP patients. Because of its well-known safety profile and low cost compared to other potential second-line treatments for ITP, a trial course of dapsone should be viewed as an attractive option before splenectomy in steroid-dependent of refractory adult ITP patients.
在免疫性血小板减少性紫癜（ITP）成年患者中，类固醇通常被作为一线治疗方案，但长期完全缓解的患者不超过20%。对于其余患者，脾切除术被视为首选治疗方法，据报道“治愈”率为60%-70%。然而，脾切除术后存在手术固有风险和败血症风险，且不能保证成功，这使得人们有理由寻求避免脾切除术的策略。在此，我们回顾性评估了达普颂治疗一线类固醇治疗失败的ITP患者的结果。这些患者在考虑脾切除术之前，接受达普颂100毫克/天治疗至少30天。疗效定义为血小板计数持续升高（>50×10⁹/升），且明显归因于达普颂治疗。在52例依赖类固醇或难治性患者中，达普颂治疗后，经中位数21.10个月的随访，44.2%的患者血小板计数持续升高。“有反应”的患者在随访中均无需进行脾切除术，而“无反应”的患者这一比例为69.0%，这一观察结果进一步证实了达普颂在这种情况下的长期疗效。与达普颂相关的不良事件较轻，停药后可迅速逆转。我们的回顾性分析结果表明，对于依赖类固醇或难治性ITP患者，达普颂是一种安全有效的二线药物。由于其安全性众所周知，且与ITP其他潜在二线治疗相比成本较低，对于依赖类固醇或难治性成年ITP患者，在进行脾切除术之前，达普颂试验疗程应被视为一个有吸引力的选择。
