Eddou Hicham, Zinebi Ali, Maaroufi Hicham El, Moudden Mohammed Karim, Doghmi Kamal, Mikdame Mohammed, Baaj Mohammed El
Service de Médecine Interne, Hôpital Militaire Moulay Ismail Méknes, Maroc.
Service d'Hématologie Clinique, Hôpital Militaire d'Instruction Mohammed V, Rabat, Maroc.
Pan Afr Med J. 2017 Oct 19;28:160. doi: 10.11604/pamj.2017.28.160.11885. eCollection 2017.
Primarye systemic AL amyloidosis is a rare hematologic disorder. The majority of the therapeutic guidelines are based on phase II studies or on retrospective comparisons and case series. Our study aimed to describe all the cases of primary AL amyloidosis reported in 2 military hospitals and to make a comparison between standard melphalan-dexamethasone protocol and new agents in first-line treatment of patients with this disease. We conducted a retrospective, descriptive and multicentric study of all patients with AL amyloidosis whose data were collected during the period July 2009-June 2016. Twenty five patients were enrolled in the study (12 patients treated with melphalan-dexamethasone and 13 with bortezomib-based protocol or lenalidomide-based protocol). There was no significant difference in the epidemiological, clinical and prognostic features between the 2 groups. After a median follow up of 40 months, median overall survival was 54 months in the melphalan-dexamethasone-treated group and 60 months in the new therapies-treated group (P = 0.98). Progression-free survival was 18 months in the standard treatment group vs 11 months in the 2nd group (p = 0.08). In our small case series we haven't found a superiority of the new therapies compared to the standard protocol. This result should be confirmed by a true prospective study, mainly because of the cost of these new molecules that are not always accessible, especially in developing countries.
原发性系统性 AL 淀粉样变性是一种罕见的血液系统疾病。大多数治疗指南基于 II 期研究、回顾性比较或病例系列。我们的研究旨在描述两家军队医院报告的所有原发性 AL 淀粉样变性病例,并比较标准美法仑 - 地塞米松方案与新药物在该疾病患者一线治疗中的效果。我们对 2009 年 7 月至 2016 年 6 月期间收集数据的所有 AL 淀粉样变性患者进行了一项回顾性、描述性多中心研究。25 名患者纳入研究(12 名患者接受美法仑 - 地塞米松治疗,13 名患者接受基于硼替佐米或来那度胺的方案治疗)。两组在流行病学、临床和预后特征方面无显著差异。中位随访 40 个月后,美法仑 - 地塞米松治疗组的中位总生存期为 54 个月,新疗法治疗组为 60 个月(P = 0.98)。标准治疗组的无进展生存期为 18 个月,第二组为 11 个月(p = 0.08)。在我们的小病例系列中,我们未发现新疗法比标准方案更具优势。这一结果应由一项真正的前瞻性研究证实,主要是因为这些新分子的成本较高,在发展中国家并非总能获得。