A Prospective, Non-interventional, Multinational Study Evaluating the Efficacy and the Safety of High Dose Therapy Followed by Autologous Hematopoietic Stem Cell Transplantation as a Frontline Therapy for Myeloma Patients With Severe Renal Impairment (IRMYG Study)
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 50
- 试验地点
- 33
- 主要终点
- Non Relapse Mortality post-transplantation
研究概览
简要总结
Multiple myeloma (MM) is a malignant plasma cell disorder, characterized by the presence of more than 10 % of clonal plasma cells in the bone marrow. Therapeutic intervention is recommended when at least one of the myeloma defining events occurs (CRAB features). Renal impairment (RI) is one of the most common complications of MM, accounting for 20-30 % of MM patients at diagnosis and 40-50% of patients during the course of their disease. To date, there is no defined consensus for the management of myeloma patients with renal failure. It is then of clinical importance to better considering available therapeutic options to improve responses and survival of these patients.
详细描述
RI is associated with poor prognosis and short median survival (32 months vs 55 months for MM patients with normal renal function). Thus, RI remains a major challenge for hematologists, including decisions on optimal anti-myeloma therapy, potential dialysis, supportive care and quality of life. The combination of a proteasome inhibitor and an immunomodulator is the preferred induction treatment for newly diagnosed transplant-eligible MM patients. After induction, high-dose therapy with Autologous Stem Cell Transplant (ASCT) is the standard of care for these patients. However, concerns related to management of comorbidities and treatment side effects question about therapeutic options for patients with severe renal damage. Of interest, recent studies argued that high-dose therapy followed by ASCT could be a feasible and safe method for renal failure MM patients. Yet, these observations on small sample size patients groups need to be confirmed with standardized conditions. This study proposes to evaluate the efficacy and the safety of this therapeutic strategy in MM patients with severe renal impairment.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 66 Years 至 —（Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age ≤ 66 years-old
- •Patients with symptomatic, measurable and newly diagnosed multiple myeloma associated:
- •Severe renal failure at the time of transplantation (creatinine clearance < 40 ml/min/1.73m², CKD-EPI: Chronic Kidney Disease Epidemiology Collaboration)
- •Partial response after induction treatment
- •For patients who undergo autologous transplantation, absence of known contraindication for transplantation
- •Absence of amylose
- •Patient affiliated to a social security regimen or beneficiary of the same
- •Signed written informed consent form
排除标准
- •Patient without at least a partial hematological response following the induction stage
- •Medical history of previous malignancy
- •Patient under guardianship or deprived of his liberty or any condition that may affect the patient's ability to understand and sign the informed consent (art. L.1121-6, L.112-7, L.1211-8, L.1211-9)
- •Pregnant or breastfeeding woman
- •Declining participation
结局指标
主要结局
Non Relapse Mortality post-transplantation
时间窗: 100 days post-transplantation
Non-relapse mortality at Day +100 post-transplantation will be reported.
次要结局
- progression-free survival(2 years post-transplantation)
- Number of toxicities(2 years post-transplantation)
- Overall survival(2 years post-transplantation)
- presence of hematological response(6 months)
- Level of renal response(3 months, 6 months and one year)