Stem Cell Transplantation for Acute Leukemia

Recruiting at 2 trial locations

Overseen By Roni Tamari, MD

Age: 18+

Sex: Any

Trial Phase: Academic

Sponsor: Memorial Sloan Kettering Cancer Center

Disqualifiers: Polycythemia vera, Essential thrombocytosis

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Approved in 4 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

1365 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores why some patients with acute leukemia or certain blood disorders, such as MDS (a bone marrow disorder) and MPN (a group of blood cancers), do or do not receive a stem cell transplant. Researchers aim to understand how many patients undergo a transplant and the reasons behind their decisions. The study includes patients with newly diagnosed or relapsed acute leukemia, or those who did not respond to initial treatments. Participants will be monitored to determine why they might choose or decline a transplant, even when recommended by their doctors. As an unphased study, this trial offers patients the chance to contribute to important research that could enhance decision-making processes for future patients considering stem cell transplants.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that using stem cells from a donor to replace damaged bone marrow is generally safe for treating certain blood cancers, such as acute leukemia. The safety of this procedure can vary based on the type of donor. Donors who are a close match, like siblings, often lead to the best results, but other donor types also show good outcomes, especially for older patients.

One study highlighted that the rates of serious side effects within the first 100 days after the transplant were similar to those seen in past treatments. This suggests that while there are risks, they are manageable and not unusually high compared to similar procedures.

Although the procedure is complex and carries some risks, existing data supports its safety for many patients with acute leukemia. ^{1 2 3 4 5}

Why are researchers excited about this trial?

Researchers are excited about this trial because it aims to uncover the hidden barriers preventing patients with acute leukemia, MDS, or MPN from receiving allogeneic hematopoietic stem cell transplantation (HCT) when they are eligible for it. This trial is unique because it focuses on understanding real-world challenges and obstacles, like logistical issues or patient hesitance, that might hinder access to this potentially life-saving treatment. By identifying these barriers, the trial hopes to improve access and outcomes for patients who can benefit from HCT, ultimately leading to better survival rates and quality of life.

What evidence suggests that Allogeneic Hematopoietic Stem Cell Transplantation might be an effective treatment for blood cancer?

Research has shown that allogeneic hematopoietic stem cell transplantation can effectively treat acute leukemia and similar blood disorders. In this trial, participants will be grouped into different arms based on their specific conditions. For older patients with acute myeloid leukemia (AML), this treatment increased one-year survival rates to 77.9%, compared to 62% with standard chemotherapy. It also significantly reduced the chance of the disease returning, from 56.6% to 16.5%. In patients with myelodysplastic syndromes (MDS) and myeloproliferative neoplasms (MPN), the three-year overall survival rate was 48.5%. This evidence suggests that this type of stem cell transplant can improve survival for patients with these conditions. ^{1 3 6 7 8}

Who Is on the Research Team?

Roni Tamari, MD

Principal Investigator

Memorial Sloan Kettering Cancer Center

Are You a Good Fit for This Trial?

Inclusion Criteria

Patients with newly diagnosed or relapsed acute leukemia. Patients undergoing reinduction due to primary induction failure are also eligible. Patients with acute leukemia will be enrolled to Arm A

Clinically significant cytopenia of at least 2 cell lines affected; Hgb<10

Platelet<100,000, absolute neutrophil count<1000

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Observation

Follow patients throughout their journey and identify prospectively the reasons why patients did not proceed to HCT when deemed appropriate and eligible

3 years

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Allogeneic Hematopoietic Stem Cell Transplantation

How Is the Trial Designed?

Group I: Patients with MDS and MPN (Arm B) Experimental Treatment 1 Intervention

Follow patients throughout their journey and identify prospectively the reasons why patients did not proceed to HCT when deemed appropriate and eligible.

Group II: Patients with Acute Leukemia or MDS/MPN who Relapse After First Allografts (Arm C) Experimental Treatment 1 Intervention

Follow patients throughout their journey and identify prospectively the reasons why patients did not proceed to HCT when deemed appropriate and eligible.

Group III: Patients with Acute Leukemia (Arm A) Experimental Treatment 1 Intervention

Follow patients throughout their journey and identify prospectively the reasons why patients did not proceed to HCT when deemed appropriate and eligible.

Allogeneic Hematopoietic Stem Cell Transplantation is already approved in European Union, United States, Canada, Japan for the following indications:

Approved in European Union as Allogeneic Hematopoietic Stem Cell Transplantation for:

• Primary immunodeficiency disorders
• Immune dysregulatory disorders
• Hemophagocytic lymphohistiocytosis
• Bone marrow failure syndromes
• Hemoglobinopathies

Approved in United States as Allogeneic Hematopoietic Stem Cell Transplantation for:

• Primary immunodeficiency disorders
• Immune dysregulatory disorders
• Hemophagocytic lymphohistiocytosis
• Bone marrow failure syndromes
• Hemoglobinopathies

Approved in Canada as Allogeneic Hematopoietic Stem Cell Transplantation for:

• Primary immunodeficiency disorders
• Immune dysregulatory disorders
• Hemophagocytic lymphohistiocytosis
• Bone marrow failure syndromes
• Hemoglobinopathies

Approved in Japan as Allogeneic Hematopoietic Stem Cell Transplantation for:

• Primary immunodeficiency disorders
• Immune dysregulatory disorders
• Hemophagocytic lymphohistiocytosis
• Bone marrow failure syndromes
• Hemoglobinopathies

Who Is Running the Clinical Trial?

Memorial Sloan Kettering Cancer Center

Lead Sponsor

Trials

1,998

Recruited

602,000+

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12533790/

Outcomes of Allogeneic Hematopoietic Stem Cell ... Haploidentical HSCT improved one-year survival (77.9% vs. 62.0% with chemotherapy) in elderly AML, with lower relapse (16.5% vs. 56.6%). Matched ...

2. nature.com nature.com/articles/s41598-025-05602-z

Clinical outcomes of allogeneic hematopoietic stem cell ... This study emphasizes the efficacy of HSCT in the treatment of infant AML, with higher OS rates compared to childhood AML. It also supports UCB as a viable ...

3. astctjournal.org astctjournal.org/article/S1083-8791(20)30312-8/fulltext

Outcome of Allogeneic Hematopoietic Stem Cell ... The 3-year overall survival (OS) was 48.5% in MDS/MPN-U patients undergoing allo-HSCT. •. Age and disease status were significantly associated with OS of MDS/ ...

4. ashpublications.org ashpublications.org/bloodadvances/article/9/20/5234/546384/Cost-effectiveness-of-upfront-vs-delayed

Cost-effectiveness of upfront vs delayed allogeneic ... The ETAL-1 trial demonstrated that upfront allogeneic hematopoietic stem cell transplant (HSCT) improved disease-free survival, but not overall ...

5. tandfonline.com tandfonline.com/doi/full/10.1080/10428194.2025.2514662

Optimal timing of allogeneic hematopoietic stem cell ... The study demonstrated a clear survival advantage for HSCT, with a 4-year overall survival rate of 37% in the transplant group compared to 20% in the non- ...

6. ascopubs.org ascopubs.org/doi/10.1200/JCO.2024.42.16_suppl.6516

Safety outcomes in patients with acute myeloid leukemia ... The use of GO appears to be safe prior to HSCT in adults with AML. Rates for 100-day VOD and TRM were comparable to those previously reported.

7. sciencedirect.com sciencedirect.com/science/article/pii/S2531137925001555

OUTCOMES OF ALLOGENEIC STEM CELL ... This study presents data on allogeneic HSCT procedures performed in our transplant center for AML patients.

8. ashpublications.org ashpublications.org/blood/article/144/Supplement%201/3552/529953/Impact-of-Minimal-Residual-Disease-Status-on

Impact of Minimal Residual Disease Status on Outcomes after ... This study aimed to assess how MRD status at the time of transplantation influences post-transplant outcomes in AML patients. METHODS: Patients ...