Myelodysplastic neoplasm with low blasts

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ORPHA:98826D46.7
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3FDA treatments7Active trials43Specialists8Treatment centers1Financial resources

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UniteRare data is sourced from FDA.gov, ClinicalTrials.gov, Orphanet, OMIM, and NORD.
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Overview

Also known as:

Clinical phenotype terms— hover any for plain English:

Anemia of inadequate productionHP:0010972Macrocytic anemiaHP:0001972Erythroid hypoplasiaHP:0012133Single lineage myelodysplasiaHP:0012150Normochromic anemiaHP:0001895Normocytic anemiaHP:0001897Bone marrow hypocellularityHP:0005528Abnormal cardiac ventricular functionHP:0030872
Orphanet ↗NORD ↗

FDA & Trial Timeline

10 events
Mar 2026Research on YTS109 Cell in Patients With Recurrent/Refractory Autoimmune Hemolytic Anemia

China Immunotech (Beijing) Biotechnology Co., Ltd. — PHASE1

TrialNOT YET RECRUITING
Mar 2026Orelabrutinib in the Treatment of Relapsed/Refractory AIHA

Peking Union Medical College Hospital — PHASE2

TrialNOT YET RECRUITING
Feb 2026Quality of Life-Guided Transfusion in Refractory MDS or AML

Centre Hospitalier Universitaire de Nice — NA

TrialRECRUITING
Jan 2026An Open-label, Single-arm, Prospective, Multicenter, Phase I/II Clinical Study on the Safety and Efficacy of CD19/BCMA CAR-T Cell Therapy for Relapsed/Refractory Warm Antibody Autoimmune Hemolytic Anemia

Yihao Wang — PHASE1, PHASE2

TrialNOT YET RECRUITING
Dec 2025A Study of YTS109 Cell in Subjects With Relapsed/Refractory Autoimmune Hemolytic Anemia

Union Hospital, Tongji Medical College, Huazhong University of Science and Technology — PHASE1

TrialNOT YET RECRUITING
Sep 2025A Single-arm Phase 2 Prospective Clinical Study of Enatumab in the Treatment of Relapsed/Refractory Warm Antibody Autoimmune Hemolytic Anemia

Bing Han — PHASE2

TrialNOT YET RECRUITING
Sep 2025A Single-arm Phase 2 Prospective Clinical Study of Linprixel in the Treatment of Relapsed/Refractory Autoimmune Hemolytic Anemia

Peking Union Medical College Hospital

TrialNOT YET RECRUITING
Jul 2025Efficacy of Immediate Allogeneic Hematopoietic Stem Cell Transplantation Versus Bridging Therapy Followed by Transplantation in Higher-Risk Myelodysplastic Syndrome Patients

Institute of Hematology & Blood Diseases Hospital, China — PHASE2

TrialRECRUITING
Jul 2025Study of Individuals and Families With Aberrations in DDX41 or Similar Cancer Predisposition Variants

National Cancer Institute (NCI)

TrialRECRUITING
Apr 2025UCAR T-cell Therapy Targeting CD19/BCMA in Patients With r/r Autoimmune Hemolytic Anemia

Zhejiang University — EARLY_PHASE1

TrialNOT YET RECRUITING

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

Treatments

3 available

Azacitidine

AZACITIDINE· Dr. Reddy's Laboratories Inc.

indicated for treatment of adult patients with refractory anemia (RA)

Azacitidine

AZACITIDINE FOR· Pilnova Pharma Inc

indicated for the treatment of adult patients with refractory anemia (RA)

Reblozyl

LUSPATERCEPT· Celgene Corporation
REBLOZYL is indicated for the treatment of anemia failing an erythropoiesis stimulating agent and requiring 2 or more red blood cell units over 8 weeks in adult patients with very low- to intermediate

REBLOZYL is indicated for the treatment of anemia failing an erythropoiesis stimulating agent and requiring 2 or more red blood cell units over 8 weeks in adult patients with very low- to intermediate-risk myelodysplastic syndromes with ring sideroblasts (MDS-RS)

Clinical Trials

7 recruitingView all trials with filters →
Phase 22 trials
Efficacy of Immediate Allogeneic Hematopoietic Stem Cell Transplantation Versus Bridging Therapy Followed by Transplantation in Higher-Risk Myelodysplastic Syndrome Patients
Phase 2
Actively Recruiting
· Sites: Beijin, Beijing Municipality; Zhengzhou, Henan +3 more · Age: 1899 yrs
Efficacy and Safety of Luspatercept for the Treatment of Anemia Due to MDS With del5q, Refractory/Resistant/Intolerant to Prior Treatments, RBC-TD
Phase 2
Actively Recruiting
· Sites: Alessandria; Ancona +21 more · Age: 1899 yrs
Phase 11 trial
A Study of TSC-100 and TSC-101 in AML, ALL and MDS in Patients Undergoing Allogeneic Peripheral Blood Stem Transplantation
Phase 1
Actively Recruiting
PI: Michelle Matzko, MD (Tscan Therapeutics) · Sites: Duarte, California; New Haven, Connecticut +13 more · Age: 1899 yrs
N/A1 trial
Quality of Life-Guided Transfusion in Refractory MDS or AML
N/A
Actively Recruiting
PI: Thomas Cluzeau (Centre Hospitalier Universitaire de Nice) · Sites: Nice · Age: 1899 yrs
Other3 trials
Study of MGUS, Smoldering Myeloma, Early MDS and CLL to Assess Molecular Events of Progression and Clinical Outcome
Actively Recruiting
PI: Irene Ghobrial, MD (Dana-Farber Cancer Institute) · Sites: Boston, Massachusetts; Brighton, Massachusetts +5 more · Age: 1899 yrs
Study of Individuals and Families With Aberrations in DDX41 or Similar Cancer Predisposition Variants
Actively Recruiting
PI: Sung-Yun Pai, M.D. (National Cancer Institute (NCI)) · Sites: Bethesda, Maryland · Age: 0120 yrs
Collection of Samples of Bone Marrow Aspiration From Patients With Myelodysplastic Syndrome
Active
· Sites: Jerusalem, Israel · Age: 1899 yrs

Specialists

Showing 25 of 43View all specialists →
SP
Stine Ulrik Mikkelsen, MD, PhD
Specialist
PI on 1 active trial
KM
Kirsten Grønbæk, Prof., MD
Specialist
PI on 1 active trial
AM
Ali Al-Mousawi, MD
Specialist
PI on 1 active trial
HM
Harinder Gill, MD
FRESNO, CA
Specialist
PI on 5 active trials
EG
Elizabeth Griffiths
SAN FRANCISCO, CA
Specialist
PI on 2 active trials
AM
Andréa TOMA, MD
Specialist
PI on 1 active trial
JP
John Pimanda, Professor
Specialist
PI on 1 active trial
FM
François Dreyfus, MD
Specialist
PI on 1 active trial
CM
Charles A. Linker, MD
SAN FRANCISCO, CA
Specialist
PI on 2 active trials
JS
Jun Shi
Specialist
PI on 3 active trials172 Myelodysplastic neoplasm with low blasts publications
AE
Anoop Enjeti
Specialist
PI on 1 active trial
TM
Thomas G. Martin, MD
Specialist
PI on 4 active trials
HM
Hannah Choe, MD
Specialist
PI on 3 active trials
GM
Guillermo Bravo, MD
CHANDLER, AZ
Specialist
PI on 1 active trial
WM
Willis Navarro, MD
TUCSON, AZ
Specialist
PI on 4 active trials
AV
Amit K Verma
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
SP
Sophie Park
Specialist
PI on 1 active trial
LM
Liwei Fang, MD
Tianjin, Tianjin Municipality
Specialist

Rare Disease Specialist

PI on 2 active trials1 Myelodysplastic neoplasm with low blasts publication

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

Financial Resources

1 resources
Reblozyl(LUSPATERCEPT)Celgene Corporation

Travel Grants

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

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about Myelodysplastic neoplasm with low blasts

Disease timeline:

New recruiting trial: Quality of Life-Guided Transfusion in Refractory MDS or AML

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

New recruiting trial: Efficacy and Safety of Luspatercept for the Treatment of Anemia Due to MDS With del5q, Refractory/Resistant/Intolerant to Prior Treatments, RBC-TD

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

New trial: A Study of Avutometinib for People With Solid Tumor Cancers

Phase PHASE1 trial recruiting. Avutometinib

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 low blasts

Are there clinical trials for Myelodysplastic neoplasm with low blasts?

Yes — 7 recruiting clinical trials are currently listed for Myelodysplastic neoplasm with low 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 low blasts?

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