Phase 2
N=50
Chemotherapy in Treating Patients With Myelodysplastic Syndrome Before Donor Stem Cell Transplant
Chronic Myelomonocytic Leukemia · de Novo Myelodysplastic Syndrome · Myelodysplastic Syndrome · Secondary Myelodysplastic Syndrome
Bottom Line
View on ClinicalTrials.gov: NCT01812252 ↗Enrolled (actual)
50
Serious AEs
—
Results posted
Feb 2024
Primary outcome: Primary: Failure-free Survival (Failure Defined as Death or Relapse) — 6; 10 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Azacitidine (AZC) (Drug); Decitabine (Drug); Quality-of-Life Assessment (Other)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Fred Hutchinson Cancer Center
- Primary completion
- Oct 2022
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Failure-free Survival (Failure Defined as Death or Relapse) |
6; 10 | — |
| SECONDARY Quality of Life Will be Assessed Using the European Organization for Research and Treatment of Cancer Quality of Life (QoL) Questionnaire (EORTC QLQ-C30) Questionnaire. |
2.27; 1.72; 2.14; 2.0; 2.0; 1.81 | — |
| SECONDARY Quality of Life Will be Assessed Using the EORTC QLQ-HDC29 a Supplementary Module Assessing the Quality of Life During and After High-dose Chemotherapy and Stem Cell Transplantation. |
1.36; 1.08; 1.2; 1.0; 1.36; 1.62 | — |
| SECONDARY Overall Survival |
7; 10; 2; 1; 5; 3 | — |
| SECONDARY Number of Patients Who Relapse Post-transplant |
6; 3; 6; 10 | — |
| SECONDARY Number of Participants Who Received a Hematopoietic Cell Transplantation (HCT). |
12; 13; 13; 12 | — |
Summary
This randomized clinical trial studies different chemotherapies in treating patients with myelodysplastic syndrome before donor stem cell transplant. Giving chemotherapy before a donor stem cell transplant helps stop the growth of cancer cells in the bone marrow, including normal blood-forming cells (stem cells) and cancer cells, and may prevent the myelodysplastic syndrome from coming back after the transplant. When the healthy stem cells from a donor are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets.
Eligibility Criteria
Inclusion Criteria
- Diagnosis of de novo or secondary myelodysplastic syndrome (MDS), including chronic myelomonocytic leukemia, as defined by the 2008 World Health Organization classification system
- Patients must have measurable disease requiring cytoreduction, defined as a bone marrow myeloblast count >= 5% and < 20% on morphologic examination or by flow cytometry in cases in which adequate morphologic examination is not possible
- Patients must be considered to have an acceptable risk of early mortality with intensive chemotherapy as determined by the attending physician at the time of the initial visit; since the specific therapy within each arm will be determined after randomization, there is no threshold of organ dysfunction or performance status for inclusion
- Considered a potential transplant candidate; the attending/treating physician will determine transplant candidacy at the time of consent
- Capable of understanding the investigational nature, potential risks and benefits of the study, and able to provide valid informed consent
Exclusion Criteria
- A diagnosis of acute promyelocytic leukemia as defined by the 2008 World Health Organization classification system
- Previous treatment for MDS or acute myeloblastic leukemia (AML) with intensive chemotherapy regimen (induction chemotherapy) or hypomethylating agent
- Have any other severe concurrent disease, or have a history of serious organ dysfunction or disease involving the heart, kidney, liver, or other organ system that may place the patient at undue risk to undergo treatment
- Patients with a systemic fungal, bacterial, viral, or other infection not controlled (defined as exhibiting ongoing signs/symptoms related to the infection and without improvement, despite appropriate antibiotics or other treatment)
- Females who are pregnant or breastfeeding
- Fertile men and women unwilling to use contraceptive techniques during and for 12 months following treatment
- Any uncontrolled or significant concurrent disease, illness, or psychiatric disorder that would compromise patient safety or compliance, interfere with consent, study participation, follow up, or interpretation of study results
- Clinical evidence suggestive of central nervous system (CNS) involvement with MDS unless a lumbar puncture confirms the absence of leukemic blasts in the cerebrospinal fluid (CSF)
Data sourced from ClinicalTrials.gov (NCT01812252). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.