Myelodysplastic neoplasm with increased blasts

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2FDA treatments11Active trials27Specialists8Treatment centers

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Overview

Also known as:

MDSMDS with excess blastsMDS-IBMyelodysplastic syndromesRAEBRefractory anemia with excess blasts

Clinical phenotype terms— hover any for plain English:

Anemia of inadequate productionHP:0010972Anemic pallorHP:0001017Retinal hemorrhageHP:0000573Acute myeloid leukemiaHP:0004808Bone marrow hypocellularityHP:0005528Abnormal bone marrow cell morphologyHP:0005561Abnormal circulating protein concentrationHP:0010876Abnormal circulating albumin concentrationHP:0012116Dysplastic granulopoesisHP:0012136Multiple lineage myelodysplasiaHP:0012148Single lineage myelodysplasiaHP:0012150
Orphanet ↗NORD ↗

FDA & Trial Timeline

10 events
Dec 2025Luspatercept + Darbepoetin in MDS

Yale University — PHASE2

TrialRECRUITING
Oct 2025Safety and Feasibility of Sulforaphane to Promote Early Haematopoietic Recovery After Cord Blood Transplantation

Institute of Hematology & Blood Diseases Hospital, China — NA

TrialRECRUITING
Sep 2025Evaluation of Treosulfan Versus Melphalan Conditioning Followed by PTCy in Patients With AML and MDS Undergoing Allogeneic Transplantation

Technische Universität Dresden — PHASE2

TrialNOT YET RECRUITING
Jan 2025A Phase Ib Study of Rezatapopt in Combination With Azacitidine in Patients With TP53Y220C Mutant Myeloid Malignancies (Acute Myeloid Leukemia or Myelodysplastic Syndrome)

M.D. Anderson Cancer Center — PHASE1

TrialRECRUITING
Jan 2024Preemptive CIML NK Cell Therapy After Hematopoietic Stem Cell Transplantation

Dana-Farber Cancer Institute — PHASE1

TrialRECRUITING
Aug 2023Treosulfan-Based Versus Clofarabine-Based Conditioning Before Donor Hematopoietic Stem Cell Transplant for the Treatment of Myelodysplastic Syndromes or Acute Myeloid Leukemia

Fred Hutchinson Cancer Center — PHASE2

TrialACTIVE NOT RECRUITING
Dec 2021Repurposing Metformin As a Leukemia-preventive Drug in CCUS and LR-MDS

Kirsten Grønbæk — PHASE2

TrialRECRUITING
Dec 2021Anti-NKG2A Monoclonal Antibody for AML or MDS Patients Undergoing Haploidentical Transplantation

Istituto Clinico Humanitas — PHASE2

TrialRECRUITING
Oct 2020ATG Plus PTCy vs ATG for CGVHD Prophylaxis

McMaster University — PHASE2

TrialACTIVE NOT RECRUITING
Jul 2020A Phase Ib Study of APG-115 Single Agent or in Combination With Azacitidine or Cytarabine in Patients With AML and MDS.

Ascentage Pharma Group Inc. — PHASE1

TrialRECRUITING

Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Treatments

2 available

Azacitidine

AZACITIDINE· Dr. Reddy's Laboratories Inc.

indicated for treatment of adult patients with refractory anemia with excess blasts (RAEB)

View Details →FDA Label ↗

Azacitidine

AZACITIDINE FOR· Pilnova Pharma Inc

indicated for the treatment of adult patients with refractory anemia with excess blasts (RAEB)

View Details →FDA Label ↗

Clinical Trials

11 recruitingView all trials with filters →
Phase 31 trial
A Study to Compare the Efficacy and Safety of Luspatercept (ACE-536) Versus Epoetin Alfa for the Treatment of Anemia Due to IPSS-R Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes (MDS) Participants Who Require Red Blood Cell Transfusions and Are ESA Naïve
Phase 3
Active
PI: Bristol-Myers Squibb (Bristol-Myers Squibb) · Sites: Berkeley, California; San Diego, California +224 more · Age: 18–99 yrs
View on ClinicalTrials.gov ↗I'm interested →
Phase 23 trials
Repurposing Metformin As a Leukemia-preventive Drug in CCUS and LR-MDS
Phase 2
Actively Recruiting
PI: Kirsten Grønbæk, Professor, MD (Rigshospitalet, Denmark) · Sites: Copenhagen, Copenhagen N · Age: 18–99 yrs
View on ClinicalTrials.gov ↗I'm interested →
Allogeneic Hematopoietic Stem Cell Transplant for GATA2 Mutations
Phase 2
Actively Recruiting
PI: Danielle E Pregent-Arnold, M.D. (National Cancer Institute (NCI)) · Sites: Bethesda, Maryland · Age: 6–70 yrs
View on ClinicalTrials.gov ↗I'm interested →
Treosulfan-Based Versus Clofarabine-Based Conditioning Before Donor Hematopoietic Stem Cell Transplant for the Treatment of Myelodysplastic Syndromes or Acute Myeloid Leukemia
Phase 2
Active
PI: Phuong Vo (Fred Hutch/University of Washington Cancer Consort) · Sites: Seattle, Washington · Age: 18–70 yrs
View on ClinicalTrials.gov ↗I'm interested →
Phase 15 trials
A Phase Ib Study of Rezatapopt in Combination With Azacitidine in Patients With TP53Y220C Mutant Myeloid Malignancies (Acute Myeloid Leukemia or Myelodysplastic Syndrome)
Phase 1
Actively Recruiting
PI: Courtney DiNardo, MD (M.D. Anderson Cancer Center) · Sites: Houston, Texas · Age: 18–99 yrs
View on ClinicalTrials.gov ↗I'm interested →
A Phase Ib Study of APG-115 Single Agent or in Combination With Azacitidine or Cytarabine in Patients With AML and MDS.
Phase 1
Actively Recruiting
PI: Jianxiang Wang, M.D. (Blood Diseases Hospital Chinese Academy of Medical) · Sites: Beijing, Beijing Municipality; Guangzhou, Guangdong +12 more · Age: 18–99 yrs
View on ClinicalTrials.gov ↗I'm interested →
Talazoparib for Cohesin-Mutated AML and MDS With Excess Blasts
Phase 1
Active
PI: Jacqueline Garcia, MD (Dana-Farber Cancer Institute) · Sites: Boston, Massachusetts; Boston, Massachusetts · Age: 18–99 yrs
View on ClinicalTrials.gov ↗I'm interested →
Study of Gene Modified Donor T Cell Infusion in Patients With Recurrent Disease After Allogeneic Transplant
Phase 1
Active
PI: Bellicum Pharmaceuticals (Bellicum Pharmaceuticals, Inc.) · Sites: Atlanta, Georgia; Westwood, Kansas +3 more · Age: 18–65 yrs
View on ClinicalTrials.gov ↗I'm interested →
Preemptive CIML NK Cell Therapy After Hematopoietic Stem Cell Transplantation
Phase 1
Actively Recruiting
PI: Roman Shapiro, MD (Dana-Farber Cancer Institute) · Sites: Boston, Massachusetts; Boston, Massachusetts · Age: 18–99 yrs
View on ClinicalTrials.gov ↗I'm interested →
Other1 trial
Collecting and Storing Blood, Bone Marrow, and Other Samples From Patients With Acute Leukemia, Chronic Leukemia, or Myelodysplastic Syndromes
Active
PI: Michael Caligiuri, MD (Ohio State University Comprehensive Cancer Center) · Sites: Mountain View, California; Pismo Beach, California +140 more · Age: 18–99 yrs
View on ClinicalTrials.gov ↗I'm interested →

Specialists

Showing 25 of 27View all specialists →
NM
Naval Daver, MD
HOUSTON, TX
Specialist
PI on 8 active trials
TP
Timothy Olson, MD, PhD
Specialist
PI on 8 active trials
CD
Colleen Delaney
OLD GREENWICH, CT
Specialist
PI on 3 active trials
FG
Francis Giles
SAN ANTONIO, TX
Specialist
PI on 4 active trials
DM
David Rizzieri, MD
CHARLOTTE, NC
Specialist
PI on 7 active trials
MM
Michael Pulsipher, MD
LOS ANGELES, CA
Specialist
PI on 6 active trials
PV
Phuong Vo
Specialist
PI on 2 active trials
GR
Guillaume Richard-Carpentier
HOUSTON, TX
Specialist
PI on 1 active trial
PB
Pamela Becker
Specialist
PI on 7 active trials16 Myelodysplastic neoplasm with increased blasts publications
CM
Christen Ebens, MD
MINNEAPOLIS, MN
Specialist
PI on 2 active trials
TM
Thomas PREBET, MD
NEW HAVEN, CT
Specialist
PI on 2 active trials
MM
Matteo Della Porta, MD
Specialist
PI on 2 active trials
AF
Alessandro Flavio Ferri
LOS ANGELES, CA
Specialist
PI on 2 active trials12 Myelodysplastic neoplasm with increased blasts publications
CM
Christine Duncan, MD
Specialist
PI on 2 active trials
RS
Roman Shapiro
BOSTON, MA
Specialist
PI on 1 active trial
SF
Steven M. Fruchtman
NEW YORK, NY
Specialist
PI on 5 active trials
SL
Sonja Loges
Specialist
PI on 1 active trial
AT
Alan Tinmouth
Specialist
PI on 1 active trial
HF
Henry C. Fung
Specialist
PI on 1 active trial
MH
Mohammad M Abdul Hay
Specialist
PI on 1 active trial
AH
Aloysius Ho
Specialist
PI on 1 active trial
DP
Danielle E Pregent-Arnold
Specialist
PI on 1 active trial
LL
Laurence LEGROS
Specialist
PI on 1 active trial
DF
Dario Ferrero
Specialist
PI on 1 active trial
YZ
Yifan Zhai
Specialist
PI on 4 active trials

Treatment Centers

8 centers
🏥 NORD

Baylor College of Medicine Rare Disease Center ↗

Baylor College of Medicine

📍 Houston, TX

🏥 NORD

Stanford Medicine Rare Disease Center ↗

Stanford Medicine

📍 Stanford, CA

🔬 UDN

NIH Clinical Center Undiagnosed Diseases Program ↗

National Institutes of Health

📍 Bethesda, MD

🔬 UDN

UCLA UDN Clinical Site ↗

UCLA Health

📍 Los Angeles, CA

🔬 UDN

Baylor College of Medicine UDN Clinical Site ↗

Baylor College of Medicine

📍 Houston, TX

🔬 UDN

Harvard/MGH UDN Clinical Site ↗

Massachusetts General Hospital

📍 Boston, MA

🏥 NORD

Mayo Clinic Center for Individualized Medicine ↗

Mayo Clinic

📍 Rochester, MN

👤 Mayo Clinic Center for Individualized Medicine

🏥 NORD

UCLA Rare Disease Day Program ↗

UCLA Health

📍 Los Angeles, CA

Travel Grants

No travel grants are currently matched to Myelodysplastic neoplasm with increased blasts.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about Myelodysplastic neoplasm with increased blasts

Disease timeline:

New recruiting trial: A Phase Ib Study of APG-115 Single Agent or in Combination With Azacitidine or Cytarabine in Patients With AML and MDS.

A new clinical trial is recruiting patients for Myelodysplastic neoplasm with increased blasts

New recruiting trial: Ivosidenib and Venetoclax With or Without Azacitidine in Treating Patients With IDH1 Mutated Hematologic Malignancies

A new clinical trial is recruiting patients for Myelodysplastic neoplasm with increased blasts

New recruiting trial: A Phase Ib Study of Rezatapopt in Combination With Azacitidine in Patients With TP53Y220C Mutant Myeloid Malignancies (Acute Myeloid Leukemia or Myelodysplastic Syndrome)

A new clinical trial is recruiting patients for Myelodysplastic neoplasm with increased blasts

New recruiting trial: Preemptive CIML NK Cell Therapy After Hematopoietic Stem Cell Transplantation

A new clinical trial is recruiting patients for Myelodysplastic neoplasm with increased blasts

New recruiting trial: Romiplostim N01 for Platelet Recovery After Haploidentical HSCT

A new clinical trial is recruiting patients for Myelodysplastic neoplasm with increased blasts

New recruiting trial: Anti-NKG2A Monoclonal Antibody for AML or MDS Patients Undergoing Haploidentical Transplantation

A new clinical trial is recruiting patients for Myelodysplastic neoplasm with increased blasts

New recruiting trial: Repurposing Metformin As a Leukemia-preventive Drug in CCUS and LR-MDS

A new clinical trial is recruiting patients for Myelodysplastic neoplasm with increased blasts

New recruiting trial: Allogeneic Hematopoietic Stem Cell Transplant for GATA2 Mutations

A new clinical trial is recruiting patients for Myelodysplastic neoplasm with increased blasts

New recruiting trial: Safety and Feasibility of Sulforaphane to Promote Early Haematopoietic Recovery After Cord Blood Transplantation

A new clinical trial is recruiting patients for Myelodysplastic neoplasm with increased blasts

New recruiting trial: Luspatercept + Darbepoetin in MDS

A new clinical trial is recruiting patients for Myelodysplastic neoplasm with increased blasts

Caregiver Resources

NORD Caregiver Resources

Support, advocacy, and financial assistance for caregivers of rare disease patients.

Mental Health Support

Rare disease caregiving can be isolating. Connect with counseling and peer support.

Family & Caregiver Grants

Financial assistance programs specifically for caregivers of rare disease patients.

Social Security Disability

Learn how rare disease patients may qualify for SSDI/SSI benefits.

Common questions about Myelodysplastic neoplasm with increased blasts

Are there clinical trials for Myelodysplastic neoplasm with increased blasts?

Yes — 11 recruiting clinical trials are currently listed for Myelodysplastic neoplasm with increased blasts on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.

Which specialists treat Myelodysplastic neoplasm with increased blasts?

25 specialists and care centers treating Myelodysplastic neoplasm with increased blasts are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.