Acute panmyelosis with myelofibrosis

Last reviewed

🖨 Print for my doctorAdvocacy Hub →
ORPHA:86843C94.4
Who is this for?
Show terms as
6Active trials16Specialists8Treatment centers1Financial resources

Where are you in your journey?

ℹ
UniteRare data is sourced from FDA.gov, ClinicalTrials.gov, Orphanet, OMIM, and NORD.
Report missing data

Overview

Also known as:

Acute myelodysplasia with myelofibrosisAcute myelofibrosis

Clinical phenotype terms— hover any for plain English:

MyelofibrosisHP:0011974Abnormality of bone marrow stromal cellsHP:0012129Abnormal megakaryocyte morphologyHP:0012143Bone marrow hypercellularityHP:0031020Low back painHP:0003419Acute myeloid leukemiaHP:0004808Acute myelomonocytic leukemiaHP:0004820Bone marrow hypocellularityHP:0005528Megakaryocyte nucleus hypolobulationHP:0031385Increased micromegakaryocyte countHP:0031386Increased total lymphocyte countHP:0100827
Orphanet ↗NORD ↗

FDA & Trial Timeline

8 events
Mar 2026Efficacy and Safety of Gecacitinib Hydrochloride in Prophylaxis Acute Graft-Versus-Host Disease After Allogeneic Hematopoietic Stem Cell Transplantation in Patients With Myelofibrosis

Nanfang Hospital, Southern Medical University — NA

TrialNOT YET RECRUITING
Sep 2024Darzalex Faspro (Daratumumab and Hyaluronidase-fihj) Before Standard Desensitization and Allogeneic Peripheral Blood Stem Cell Transplantation in Adult Patients at High-risk for Primary Graft Failure Secondary to Donor Specific Antibodies

Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins — EARLY_PHASE1

TrialRECRUITING
May 2024A Telehealth Advance Care Planning Intervention

University of Rochester — NA

TrialRECRUITING
Jul 2022Combination Navitoclax, Venetoclax and Decitabine for Advanced Myeloid Neoplasms

Jacqueline Garcia, MD — PHASE1

TrialACTIVE NOT RECRUITING
Aug 2021Study of Oral Administration of LP-118 in Patients With Relapsed or Refractory CLL, SLL, MDS, MDS/MPN, AML, CMML-2, MPN-BP, ALL, MF, NHL, RT, MM or T-PLL.

Newave Pharmaceutical Inc — PHASE1

TrialRECRUITING
Dec 2020

Kalydeco: FDA approved

treatment of cystic fibrosis (CF) in patients age 4 months and older who have one mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to ivacaftor potentiation based on clinical and/or in vitro assay data

FDAcompleted
Nov 2019Triplex Vaccine in Preventing CMV Infection in Patients Undergoing Hematopoietic Stem Cell Transplantation

City of Hope Medical Center — PHASE2

TrialACTIVE NOT RECRUITING
Mar 2018Haploidentical Allogeneic Peripheral Blood Transplantation: Examining Checkpoint Immune Regulators' Expression

Dartmouth-Hitchcock Medical Center — PHASE3

TrialACTIVE NOT RECRUITING

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

Treatments

No FDA-approved treatments are currently listed for Acute panmyelosis with myelofibrosis.

6 clinical trialsare actively recruiting — trials can provide access to cutting-edge therapies.

View clinical trials →

Clinical Trials

6 recruitingView all trials with filters →
Phase 31 trial
Haploidentical Allogeneic Peripheral Blood Transplantation: Examining Checkpoint Immune Regulators' Expression
Phase 3
Active
PI: Kenneth Meehan, MD (Dartmouth-Hitchcock Medical Center) · Sites: Lebanon, New Hampshire · Age: 18–75 yrs
View on ClinicalTrials.gov ↗I'm interested →
Phase 21 trial
Triplex Vaccine in Preventing CMV Infection in Patients Undergoing Hematopoietic Stem Cell Transplantation
Phase 2
Active
PI: Ryotaro Nakamura (City of Hope Medical Center) · Sites: Duarte, California · Age: 18–99 yrs
View on ClinicalTrials.gov ↗I'm interested →
Phase 12 trials
Study of Oral Administration of LP-118 in Patients With Relapsed or Refractory CLL, SLL, MDS, MDS/MPN, AML, CMML-2, MPN-BP, ALL, MF, NHL, RT, MM or T-PLL.
Phase 1
Actively Recruiting
· Sites: Chicago, Illinois; Boston, Massachusetts +6 more · Age: 13–99 yrs
View on ClinicalTrials.gov ↗I'm interested →
Combination Navitoclax, Venetoclax and Decitabine for Advanced Myeloid Neoplasms
Phase 1
Active
PI: Jacqueline S Garcia, MD (Dana-Farber Cancer Institute) · Sites: Boston, Massachusetts; Boston, Massachusetts +1 more · Age: 18–99 yrs
View on ClinicalTrials.gov ↗I'm interested →
N/A1 trial
A Telehealth Advance Care Planning Intervention
N/A
Actively Recruiting
PI: Kah Poh Loh (University of Rochester) · Sites: Rochester, New York · Age: 60–99 yrs
View on ClinicalTrials.gov ↗I'm interested →

Specialists

16 foundView all specialists →
JM
Jorge Cortes, MD
Specialist
PI on 17 active trials
RN
Ryotaro Nakamura
Duarte, California
Specialist

Rare Disease Specialist

PI on 6 active trials
DM
Damiano Rondelli, MD
CHICAGO, IL
Specialist
PI on 4 active trials
AR
Andrew Rezvani
STANFORD, CA
Specialist
PI on 2 active trials
AA
Aref Al-Kali
ROCHESTER, MN
Specialist
PI on 4 active trials1 Acute panmyelosis with myelofibrosis publication
MM
Melhem Solh, MD
Atlanta, Georgia
Specialist

Rare Disease Specialist

PI on 3 active trials
AR
Alessandro Rambaldi
Specialist
PI on 3 active trials
KL
Kah Poh Loh
ROCHESTER, NY
Specialist
PI on 2 active trials
UP
Uday R Popat
HOUSTON, TX
Specialist
PI on 2 active trials
RI
Racquel D Innis-Shelton
BIRMINGHAM, AL
Specialist
PI on 1 active trial
CC
Christopher R. Cogle
GAINESVILLE, FL
Specialist
PI on 1 active trial
JG
Jacqueline S Garcia
Specialist
PI on 2 active trials
AB
Andrea Bacigalupo
Specialist
PI on 2 active trials
PB
Peter van Balen
Specialist
PI on 1 active trial
CG
Christian B Gocke
BALTIMORE, MD
Specialist
PI on 1 active trial
UB
Uma Borate
COLUMBUS, OH
Specialist
PI on 1 active trial

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

Kalydeco

Vertex Pharmaceuticals Incorporated

Kalydeco Patient Support (Vertex GPS)

Patient Assistance
Manufacturer Program
Accepting applications

Travel Grants

No travel grants are currently matched to Acute panmyelosis with myelofibrosis.

Search all travel grants →NORD Financial Assistance ↗

Community

Open Acute panmyelosis with myelofibrosisForum →

No community posts yet. Be the first to share your experience with Acute panmyelosis with myelofibrosis.

Start the conversation →

Latest news about Acute panmyelosis with myelofibrosis

2 articles
NewsFDA
FDA Approves KALYDECO
KALYDECO (IVACAFTOR) received FDA approval.
NewsFDA
FDA Approves KALYDECO
KALYDECO (IVACAFTOR) received FDA approval.
See all news about Acute panmyelosis with myelofibrosis →

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 Acute panmyelosis with myelofibrosis

Are there clinical trials for Acute panmyelosis with myelofibrosis?

Yes — 6 recruiting clinical trials are currently listed for Acute panmyelosis with myelofibrosis on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.

Which specialists treat Acute panmyelosis with myelofibrosis?

16 specialists and care centers treating Acute panmyelosis with myelofibrosis are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.

What treatment and support options exist for Acute panmyelosis with myelofibrosis?

1 patient support program are currently tracked on UniteRare for Acute panmyelosis with myelofibrosis. See the treatments and support programs sections for copay assistance, eligibility, and contact details.