CRISPR−Cas9 CD33-deleted allogeneic hematopoietic cell transplantation with gemtuzumab ozogamicin maintenance in AML: a phase 1/2 trial
, and no prolonged high-grade cytopenias were observed. The most common adverse events were cytopenias and infections. Three cases of transplant-related mortality were observed due to renal failure, sepsis and sinusoidal obstruction syndrome, respectively. In summary, trem-cel demonstrated safe, rapid, robust engraftment, and GO maintenance was administered without prolonged hematologic toxicity. ClinicalTrials.gov identifier: NCT04849910 .
Authors
- Brenda Cooper (ORCID: https://orcid.org/0000-0001-9376-9377)
- Sanjana Thosar (ORCID: https://orcid.org/0000-0001-5894-4176)
- Sharon L. Hyzy (ORCID: https://orcid.org/0000-0001-8359-207X)
- Muhammad Umair Mushtaq (ORCID: https://orcid.org/0000-0001-8122-0563)
- Hyung C. Suh (ORCID: https://orcid.org/0000-0001-6912-4157)
- Darren Stanizzi
- Guy Mundelboim
- Glen Raffel
- Juliana Xavier-Ferrucio (ORCID: https://orcid.org/0000-0002-1065-1750)
- Michael R. Loken (ORCID: https://orcid.org/0000-0002-0335-9065)
- Bin E. Li
- J. DiPersio
- Léa Bernard
- Deborah Lloyd
- Chad Hudson
- Christopher A. Slapak
- Guangwu Guo (ORCID: https://orcid.org/0009-0008-7594-124X)
- Huanying Gary Ge
- Joseph Rimando
- Vanessa E. Kennedy
- Joseph Maakaron
- Guenther Koehne (ORCID: https://orcid.org/0009-0008-6281-4655)
- Sagar S. Patel (ORCID: https://orcid.org/0000-0001-9949-3615)
- Michelle I. Lin (ORCID: https://orcid.org/0009-0005-9297-2855)
- Melissa M. Lee-Sundlov
- Roni Tamari (ORCID: https://orcid.org/0000-0002-2386-0850)
- Nirali N. Shah (ORCID: https://orcid.org/0000-0002-8474-9080)
- Divya Koura
Institutions
- Baptist Hospital of Miami (US)
- National Institutes of Health (US)
- University of Minnesota (US)
- Hackensack University Medical Center (US)
- Memorial Sloan Kettering Cancer Center (US)
- Emory University (US)
- Washington University in St. Louis (US)
- UC San Diego Health System (US)
- Huntsman Cancer Institute (US)
- Hôpital Maisonneuve-Rosemont (CA)
- The University of Kansas Cancer Center (US)
- Baptist Health South Florida (US)
- University Hospitals Seidman Cancer Center (US)
- Stanford Blood Center (US)
- National Cancer Institute (US)
- Center for Cancer Research (US)
- Moores Cancer Center
Publication Details
- Journal
- Nature Medicine
- Published
- 2026-05-12
- DOI
- https://doi.org/10.1038/s41591-026-04362-1
- Primary Topic
- Acute Myeloid Leukemia Research
- Type
- article
- Field-Weighted Citation Impact
- 0.00