A Study Comparing Allogeneic Hematopoietic Cell Transplantation Versus Best Available Standard of Care Therapy in Elderly Patients With Acute Myeloid Leukemia

NCT04822766 | Active Not Recruiting

• 2 other identifiers: APHP2001342020-A01456-33
• Study Type: interventional
• Target: 24
• Locations: 1 country
• Sites: 1

Brief Summary

A subject of major interest for researchers, clinicians, patients, and payers, is the role of allogeneic hematopoietic stem cell transplantation (allo-HSCT) in the treatment of these older patients with AML. With conventional induction chemotherapy or hypomethylating agents, the expected 2-year overall survival (OS) is less than 25% in patients with intermediate- or high-risk disease. The 2-year OS ranges from 50 to 56% with allo-HSCT in AML patients older than 65 years. Performing an allo-HSCT in older patients is however still controversial because of the higher risk of non-relapse mortality (15 to 35%) and graft-versus-host disease. Depending on the center policy, patients older than 65 years will either be contraindicated for transplant or will receive allo-HSCT. With a phase III comparative, randomized, controlled, prospective, multicenter study, the trial aim to assess prospectively the outcomes and quality of life of older patients with AML receiving allo-HSCT strategy compared to those receiving a non-transplant approach.

Conditions

Acute Myeloid Leukemia

Keywords

Allogeneic; hematopoietic cell transplantation; acute myeloid leukemia

Outcome Measures

Primary Outcomes (1)

• Overall survival

  From inclusion (time of identification of potential donor) until death or at 24 months, whichever comes first\]

  2 years after the inclusion

Secondary Outcomes (9)

• Leukemia free survival

  within the 2 years after inclusion

• Assessment of MRD and time to relapse from inclusion up to 2 years

  : time between inclusion and date of relapse or at 24 months, whichever comes first]

• Quality of life FACT-BMT

  at baseline, 12 and 24 months after inclusion

• Quality of life EQ 5D 5L

  at baseline, 12 and 24 months after inclusion

• The Incremental cost-effectiveness ratios (ICERs) expressed in cost per quality-adjusted life-year (QALY) gained

  2 years after inclusion

Study Arms (2)

Chemotherapy

ACTIVE COMPARATOR

Drug: Best chemotherapy treatment

Allogeneic Hematopoietic Cell Transplantation

EXPERIMENTAL

Time of transplant procedure The best available treatments of AML

Procedure: Hematopoietic stem cell transplantation

Interventions

Hematopoietic stem cell transplantation PROCEDURE

patients will undergo allo-HSCT after consolidation therapy (or completion of other appropriate non-palliative strategy) according to standard procedures of the transplant center (choice of donor, conditioning regimen, GVHD and infection prophylaxis). The use of novel therapies (such as sorafenib, midaustorine, venetoclax, etc.) will be allowed as post-transplantation maintenance strategy.

Allogeneic Hematopoietic Cell Transplantation

Best chemotherapy treatment DRUG

patients will be treated according to the standard procedures of the treating center for this type of population. Patients will receive the best available treatments (including additional conventional chemotherapy or other non-palliative therapies such as 5-azacytidine, decitabine, venetoclax, midaustorine, enasidenib, etc.).

Chemotherapy

Eligibility Criteria

• Age: 65 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Older Adult (65+)

You may qualify if:

• Men and women
• Age ≥ 65 and ≤ 75 years
• Newly diagnosed patients with de novo or secondary AML in first complete remission who are considered as potential candidates and eligible for an allo-HSCT procedure
• Presence of a donor (matched related or unrelated or haplo-mismatched) willing to donate peripheral blood stem cells
• Patient is fit for the allo-HSCT procedure
• Patient is fit for further consolidation therapy (non-transplant arm)
• Written informed consent

You may not qualify if:

• Acute promyelocytic leukemia (AML FAB M3)
• AML deemed not eligible for allo-HSCT
• Karnofsky score \<70%
• HIV positive patient
• Life expectancy less than one month according to the attending physician
• Acute or chronic heart failure (Cardiac ejection fraction \< 40%)
• Pulmonary function - diffusion capacity \< 50% predicted
• Estimated glomerular filtration rate \< 50 ml/min (CKD-EPI)
• Severe neurological disorders
• Patient subject to a legal protection measure (guardianship, curatorship and safeguard of justice) or unable to consent
• Patient deprived of their liberty by a judicial or administrative decision
• Patient with severe psychiatric disorders or hospitalized without consent for psychiatric care

Sponsors & Collaborators

• Assistance Publique - Hôpitaux de Paris: lead

Study Sites (1)

Saint Antoine Hospital - Hematology Department

Paris, 75012, France

Location

MeSH Terms

Conditions

Leukemia, Myeloid, Acute

Interventions

Hematopoietic Stem Cell Transplantation

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases

Intervention Hierarchy (Ancestors)

Stem Cell Transplantation; Cell Transplantation; Cell- and Tissue-Based Therapy; Biological Therapy; Therapeutics; Transplantation; Surgical Procedures, Operative

Study Officials

• Rémy DULERY, MD

  Assistance Publique - Hôpitaux de Paris

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 24, 2021
• First Posted: March 30, 2021
• Study Start: December 31, 2021
• Primary Completion (Estimated): June 1, 2027
• Study Completion (Estimated): June 1, 2027
• Last Updated: August 13, 2025

Record last verified: 2025-08

Locations

FR (1)