Complications after hematopoietic stem cell transplantation

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6FDA treatments3Active trials34Specialists8Treatment centers4Financial 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:

Orphanet ↗NORD ↗

FDA & Trial Timeline

10 events
Apr 2026Respiratory Microbioma and Respiratory Complications After Hematopoietic Stem Cell Transplantion

University Hospital, Caen

TrialNOT YET RECRUITING
Mar 2026APHEXDA: New indication approved
FDAcompleted
Jan 2026Critical Illness After Hematopoietic Cell Transplantation

Peking University People's Hospital

TrialNOT YET RECRUITING
Dec 2025

Yartemlea: FDA approved

treatment of adult and pediatric patients 2 years of age and older with hematopoietic stem cell transplant-associated thrombotic microangiopathy (TA-TMA)

FDAcompleted
Oct 2024Comparison of Intralipid With SMOF Lipid Following HSCT

Shahid Beheshti University — NA

TrialRECRUITING
Aug 2024Moxibustion in the Treatment of Hemorrhagic Cystitis After Allo-HSCT

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

TrialRECRUITING
May 2024Efficacy and Safety of Avatrombopag in the Treatment of Thrombocytopenia After Haplo-HSCT

Peking University People's Hospital — PHASE2, PHASE3

TrialRECRUITING
Mar 2024Moxibustion for the Prevention of Hemorrhagic Cystitis After Allo-HSCT

Yi Zhang — NA

TrialRECRUITING
Mar 2024AlloLife - Life After Transplantation

University Hospital, Essen — NA

TrialNOT YET RECRUITING
Sep 2023High-Altitude Hematology Observation-Stem Cell Transplantation (HALO-SCT)

Yigeng Cao,MD,PhD — NA

TrialRECRUITING

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

Treatments

6 available

Defitelio

defibrotide· Jazz Pharmaceuticals, Inc.
DEFITELIO is indicated for the treatment of adult and pediatric patients with hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome (SOS), with renal or pulmonary dysfunc

DEFITELIO is indicated for the treatment of adult and pediatric patients with hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome (SOS), with renal or pulmonary dysfunction following hematopoietic stem-cell transplantation (HSCT)

PREVYMIS

letermovir· Merck Sharp & Dohme LLC
Prophylaxis of cytomegalovirus (CMV) infection and disease in adult and pediatric patients 6 months of age and older and weighing at least 6 kg who are CMV-seropositive recipients [R+] of an allogenei

Prophylaxis of cytomegalovirus (CMV) infection and disease in adult and pediatric patients 6 months of age and older and weighing at least 6 kg who are CMV-seropositive recipients [R+] of an allogeneic hematopoietic stem cell transplant (HSCT)

Leukine

Sargramostim· Immunex Corporation

For the acceleration of myeloid reconstitution following autologous bone marrow or peripheral blood progenitor cell transplantation in adult and pediatric patients 2 years of age and older

Yartemlea

narsoplimab-wuug· Omeros CorporationOrphan Drug

treatment of adult and pediatric patients 2 years of age and older with hematopoietic stem cell transplant-associated thrombotic microangiopathy (TA-TMA)

Grafapex

TREOSULFAN· Medexus Pharma, Inc.■ Boxed WarningOrphan Drug
in combination with fludarabine as a preparative regimen for allogeneic hematopoietic stem cell transplantation in adult and pediatric patients 1 year of age and older with acute myeloid leukemia (AML

in combination with fludarabine as a preparative regimen for allogeneic hematopoietic stem cell transplantation in adult and pediatric patients 1 year of age and older with acute myeloid leukemia (AML); and in combination with fludarabine as a preparative regimen for allogeneic hematopoietic stem cell transplantation in adult and pediatric patients 1 year of age and older with myelodysplastic syndrome (MDS) (ODE held by Medexus Pharma, Inc.)

Aphexda

motixafortide· Ayrmid Pharma Ltd.Orphan Drug

in combination with filgrastim (G-CSF) to mobilize hematopoietic stem cells to the peripheral blood for collection and subsequent autologous transplantation in patients with multiple myeloma

Clinical Trials

3 recruitingView all trials with filters →
N/A3 trials
Comparison of Intralipid With SMOF Lipid Following HSCT
N/A
Actively Recruiting
PI: Zahra Vahdat Shariatpanahi, Professor (School of Nutrition Sciences and Food Technology, ) · Sites: Tehran · Age: 218 yrs
Moxibustion for the Prevention of Hemorrhagic Cystitis After Allo-HSCT
N/A
Actively Recruiting
· Sites: Zhengzhou, Henan; Wuhan, Hubei +2 more · Age: 1460 yrs
Moxibustion in the Treatment of Hemorrhagic Cystitis After Allo-HSCT
N/A
Actively Recruiting
· Sites: Wuhan, Hubei · Age: 1855 yrs

Specialists

Showing 25 of 34View all specialists →
XZ
Xiaohui Zhang
TAMPA, FL
Specialist
PI on 4 active trials
XM
Xiaohui Zhang, MD
Beijing, FL
Specialist

Rare Disease Specialist

PI on 12 active trials
MM
Mary Horowitz, MD, MS
CHICAGO, IL
Specialist
PI on 10 active trials
FC
Federica Cattaneo
Specialist
PI on 1 active trial5 Complications after hematopoietic stem cell transplantation publications
RM
Richard Nash, MD
Specialist
PI on 2 active trials
GM
Gregory Yanik, MD
ANN ARBOR, MI
Specialist
PI on 2 active trials1 Complications after hematopoietic stem cell transplantation publication
CM
Christa S Zerbe, M.D.
LANCASTER, PA
Specialist
PI on 5 active trials
TM
Thuy Koll, MD
OMAHA, NE
Specialist
PI on 2 active trials
NM
Natalia Maximova, MD
Specialist
PI on 2 active trials
AM
Alice Bertaina, MD
PALO ALTO, CA
Specialist
PI on 1 active trial1 Complications after hematopoietic stem cell transplantation publication
TP
Tobias Feuchtinger, Prof
Specialist
PI on 1 active trial
DM
David Gómez-Almaguer, MD
Specialist
PI on 1 active trial
MD
Martin Schmidt-Hieber, Dr
Specialist
PI on 1 active trial
AF
Anke Friedetzky
Specialist
PI on 1 active trial
XM
Xiao-Jun Huang, M.D.
Specialist
PI on 2 active trials
BP
bergeron-lafaurie Anne, MD, PhD
Specialist
PI on 1 active trial
MM
Michael Schleuning, Prof. Dr. med.
Specialist
PI on 1 active trial
PM
Paul Grimm, MD
MERRITT ISLAND, FL
Specialist
PI on 1 active trial
CD
Christina Dieli-Conwright
Specialist
PI on 2 active trials
GP
Guenther Koehne, MD, PhD
MIAMI, FL
Specialist
PI on 2 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

Financial Resources

4 resources
Defitelio(defibrotide)Jazz Pharmaceuticals, Inc.
PREVYMIS(letermovir)Merck Sharp & Dohme LLC
Leukine(Sargramostim)Immunex Corporation

Neupogen

Amgen, Inc.

Neupogen Patient Support (Amgen Safety Net)

Patient Assistance
Manufacturer Program
Accepting applications

Travel Grants

No travel grants are currently matched to Complications after hematopoietic stem cell transplantation.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about Complications after hematopoietic stem cell transplantation

5 articles
NewsTRANSPLANTATION AND CELLULAR THERAPYApr 8, 2026
A Significant Risk Factor for Post-Transplant Lymphoproliferative Disorder in Pediatric Recipients: Low CD3+CD4+ T Cell Count at 30 Days after Hematopoietic Stem Cell Transplantation.
Post-transplant lymphoproliferative disorder (PTLD) is a potentially life-threatening complication that arises early after pediatric hematopoietic stem cell tra
NewsFRONTIERS IN MEDICINEApr 6, 2026
Development and effectiveness evaluation of a mobile health-based follow-up management model for patients after hematopoietic stem cell transplantation.
To evaluate the association between mobile health (mHealth)-based follow-up and quality of life, self-management capacity, and complication control in patients
NewsTRANSPLANTATION AND CELLULAR THERAPYMar 29, 2026
Kidney and Blood Pressure Outcomes in Children Surviving at Least 6 Months after Hematopoietic Stem Cell Transplantation: A Systematic Review and Meta-Analysis.
Pediatric hematopoietic stem cell transplantation (HSCT) recipients are at risk of developing kidney and high blood pressure (BP) conditions, but the prevalence
NewsHEMATOLOGY, TRANSFUSION AND CELL THERAPYMar 29, 2026
Mineral disorders after hematopoietic stem cell transplantation in patients receiving parenteral nutrition.
Parenteral nutrition is a critical therapeutic approach for patients undergoing hematopoietic stem cell transplantation. However, parenteral nutrition is associ
NewsPEDIATRIC HEMATOLOGY AND ONCOLOGYMar 15, 2026
Long-Term Health Outcomes in Male Survivors of Childhood Cancer After Hematopoietic Stem Cell Transplantation: A Registry-Based Study.
Late effects are a major concern after pediatric hematopoietic stem cell transplantation (HSCT), but data on health outcomes beyond early adulthood remain scarc
See all news about Complications after hematopoietic stem cell transplantation

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 Complications after hematopoietic stem cell transplantation

Are there clinical trials for Complications after hematopoietic stem cell transplantation?

Yes — 3 recruiting clinical trials are currently listed for Complications after hematopoietic stem cell transplantation on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.

Which specialists treat Complications after hematopoietic stem cell transplantation?

25 specialists and care centers treating Complications after hematopoietic stem cell transplantation are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.

What treatment and support options exist for Complications after hematopoietic stem cell transplantation?

4 patient support programs are currently tracked on UniteRare for Complications after hematopoietic stem cell transplantation. See the treatments and support programs sections for copay assistance, eligibility, and contact details.