Assessment of Allogeneic Hematopoietic Cell Transplantation in Medicare Beneficiaries With Multiple Myeloma
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
- В протоколе указаны: Allogeneic Hematopoietic Stem Cell Transplant.
- Кому может быть актуально
- Состояния в реестре: Multiple Myeloma. Базовые параметры: Без ограничений · 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
- США
- 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
Assessment of Allogeneic Hematopoietic Cell Transplantation in Medicare Beneficiaries With Multiple Myeloma: A Study to Develop Evidence of Effectiveness for the Centers for Medicare and Medicaid Services (CMS)
Обзор
Multiple myeloma (MM) is the second most common hematologic malignancy in adults. The current standard of care for MM patients fit to undergo high dose conditioning chemotherapy is an autologous HCT (autoHCT). Allogeneic HCT (alloHCT) is the only potentially curative therapy available to patients with MM. However, the significant morbidity and mortality of this procedure historically limited its application in older patients. Thus, although potentially curative, standard risk MM patients have excellent prognoses in the era of novel therapies which reduces the overall benefit of alloHCT. However, because the outcomes for high-risk MM remain poor despite the best available standard therapies (overall survival of 24-36 months), initial data suggest that alloHCT should be explored in this subset.
Подробное описание
Multiple myeloma (MM) is the second most common hematologic malignancy in adults. Overall survival (OS) in MM has improved significantly in the last 15 years with the emergence of novel therapies such as thalidomide, bortezomib and lenalidomide. The median life expectancy of patients with MM treated in the current era is more than 6 years, while SEER data from a slightly earlier time period (2008-12) estimated the 5 year survival at 48.5%. However, prognosis is not uniform and varies considerably based on a presenting features and response to therapy.
The current standard of care for MM patients fit to undergo high dose conditioning chemotherapy is an autologous HCT (autoHCT). There is controversy regarding the timing of autoHCT after initial novel therapy induction with randomized trials showing similar OS whether done early or delayed to time of relapse as salvage therapy. However, more recent trials comparing early versus delayed transplant support the benefit of early upfront autoHCT.
Allogeneic HCT (alloHCT) is the only potentially curative therapy available to patients with MM. However, the significant morbidity and mortality of this procedure historically limited its application in older patients. Current data from the Center for International Blood and Marrow Research (CIBMTR) show transplant-related mortality rates of 23 (20-26)% at 5 years with myeloablative conditioning.
Thus, although potentially curative, standard risk MM patients have excellent prognoses in the era of novel therapies which reduces the overall benefit of alloHCT. However, because the outcomes for high-risk MM remain poor despite the best available standard therapies (overall survival of 24-36 months), initial data suggest that alloHCT should be explored in this subset.
Вмешательства
- Другое Allogeneic Hematopoietic Stem Cell Transplant
This observational study will compare outcomes of prospectively enrolled HCT recipients with outcomes of a cohort of matched autoHCT controls.
Первичные конечные точки
- Compare five-year survival [Срок оценки: 5 years post transplant]
Вторичные конечные точки (5)
- Progression-free survival (PFS) [Срок оценки: 5 years post transplant]
- Relapse or progression [Срок оценки: 5 years post transplant]
- Transplant related mortality [Срок оценки: 5 years post transplant]
- Incidence of acute GVHD [Срок оценки: 5 years post transplant]
- Incidence of chronic GVHD [Срок оценки: 5 years post transplant]
Критерии участия
Критерии включения
- Medicare beneficiary
- Stage II or III multiple myeloma and/or primary plasma cell leukemia
- Eligible to receive an allogeneic HCT from any suitable allogeneic donor (as determined by the transplant center) including umbilical cord blood
- Will receive allogeneic HCT at a US transplant center
- Agree to submit comprehensive clinical data on their pre- and post-transplant clinical status and outcomes to the CIBMTR
Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.
Здоровые добровольцы: Нет
Дизайн исследования
- Модель наблюдения
- Когортное
Центры проведения
США · 1 центр
- Center for International Blood and Marrow Transplant Research — Minneapolis
Идентификаторы
NCT: NCT03127761 · 17-CMS-MM