REal World MAIA UK OutcomEs
Ориентир для пациента и семьи
Простыми словами
Автоматическая сводка по структурированным данным реестра. Она помогает сориентироваться, но не заменяет официальный протокол или оценку врача.
- Что изучают
- Это наблюдательное исследование: исследуемое лечение участникам по протоколу не назначают.
- Кому может быть актуально
- Состояния в реестре: Myeloma. Базовые параметры: от 18 лет · Все.
- Что важно проверить
- Возраст, диагноз и пол — только базовые ориентиры. Предыдущее лечение, анализы и другие обязательные условия указаны ниже в критериях участия.
- Где проводится
- Великобритания
- Следующий шаг
- Сохраните исследование, покажите его лечащему врачу и уточните актуальный статус у исследовательского центра. Расходы, документы и поездка →
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Официальное название
A Retrospective Study of Clinical Outcomes in Newly Diagnosed, Transplant Ineligible Multiple Myeloma Patients Treated With Daratumumab, Lenalidomide and Dexamethasone (DRd) Outside of Clinical Trials in the UK
Обзор
This study will describe the use of triplet therapy with daratumumab, lenalidomide and dexamethasone (DRd) in the treatment of for transplant ineligible (TIE) untreated myeloma outside of clinical trials and assess the associated clinical outcomes.
Подробное описание
Triplet therapy with daratumumab, lenalidomide and dexamethasone (DRd) for transplant ineligible (TIE) untreated myeloma patients (MAIA) was reported in 2019. NICE approved this in September 2023 and since this time DRd has become the standard of care regimen for TIE patients with newly diagnosed multiple myeloma in the UK. Although there are reports of real world experience (RWE) of DRd efficacy in relapsed setting, there are no RWE reports of DRd efficacy and outcomes from the UK where it is used in the upfront setting and very limited data from Europe. Moreover, UK clinicians often adopt a pragmatic dose adjustment approach, particularly in the dosing of lenalidomide (escalation and de-escalation) with steroid tapering. As well as reducing short-term toxicities, this approach may lead to longer term benefits by reducing long-term steroid adverse effects such as steroid-induced diabetes, help ameliorate immune paresis and reduce infection risk.
However, there is very limited data on the efficacy and outcomes of this practice. In particular, there is no published RWE on the impact of pre-emptive dose modifications on tolerability and efficacy in frail patients, the cohort in which the highest treatment discontinuation rates were observed in the MAIA trial. It is also perceived that patients with comorbidities, which would have been excluded in MAIA cohort, are benefiting from this flexible approach in real world practice, especially people with chronic kidney disease and other comorbidities. A proportion of patients initially deemed fit for autologous stem cell transplantation (received D-VTD as induction) are also receiving DRd if they fail to receive a transplant. These patients were not represented in the MAIA study and the outcomes following de-escalation from D-VTD to DRd are unknown.
Первичные конечные точки
- Overall response rate (OOR) at 12 months [Срок оценки: 12 months]
- Real-world dosing strategy for DRd - starting doses of Daratumumab, Lenalidomide and dexamethasone in cycle 1 and relative dose intensity at 12 months [Срок оценки: 12 months]
Вторичные конечные точки (7)
- Progression -Free Survival (PFS) at 12 and 24 months [Срок оценки: 12 months and 24 months]
- Overall survival at 12 and 24 months [Срок оценки: 12 months and 24 months]
- Very good partial response (VGPR) [Срок оценки: 24 months]
- Occurrence of severe infections [Срок оценки: 12 months and 24 months from starting treatment]
- Treatment exposure /discontinuation (Treatment deliverability) [Срок оценки: 12 months and 24 months]
- Dosing practice and outcome difference between academic and DGH trusts [Срок оценки: 12 months and 24 months]
- Treatment setting [Срок оценки: 12 months and 24 months]
Критерии участия
Критерии включения
- Age ≥18 years
- Diagnosis of NDMM
- Not eligible for autologous stem cell transplant at diagnosis
- Received frontline DRd treatment following NICE approval (post-September 2023)
- Minimum 3 months of follow-up data available
Критерии исключения
- Participation in an interventional clinical trial for first-line therapy
- Insufficient treatment or follow-up data for analysis
- DRd used in relapsed/refractory setting rather than newly diagnosed disease
Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.
Дизайн исследования
- Модель наблюдения
- Только случаи
Центры проведения
Великобритания · 1 центр
- The Royal Wolverhampton NHS Trust — Wolverhampton
Идентификаторы
NCT: NCT07532473 · 2026HAE147