Evidence map›Paper›PMID 40523209›Full record

Trial reportJournal of clinical oncology : official journal of the American Society of Clinical Oncology2025

Post-Transplant Cyclophosphamide-Based Graft-Versus-Host Disease Prophylaxis After Mismatched Unrelated Donor Peripheral Blood Stem Cell Transplantation.

Monzr M Al Malki, Stephanie Bo-Subait, Brent Logan, Janelle Olson, Jianqun Kou, Sarah Smith, Erin Leckrone, Juan Wu, Heather E Stefanski, Jeffery J Auletta and 23 more

Erratum issued Registry-linked trialAbstract readMulticenter StudyClinical Trial, Phase II
In one paragraph

Trial report in Journal of clinical oncology : official journal of the American Society of Clinical Oncology, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. It is linked to trial NCT04904588 (A Multi-Center, Phase II Trial of HLA-Mismatched Unrelated Donor Hematopoietic Cell Transplantation With Post-Transplantation Cyclophosphamide for Patients With Hematologic Malignancies), which is not on this map. Cited by 18 papers, 2 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
18citing papers in PubMed, 2 pooled it
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

NCT04904588 phase2active not recruitingnot on this map

A Multi-Center, Phase II Trial of HLA-Mismatched Unrelated Donor Hematopoietic Cell Transplantation With Post-Transplantation Cyclophosphamide for Patients With Hematologic Malignancies

TypeinterventionalSponsorCenter for International Blood and Marrow Transplant ResearchRan2021 to 2026Enrolled300ConditionsAcute Lymphoblastic Leukemia, Acute Myelogenous Leukemia, Mixed Phenotype Acute Leukemia, Acute LeukemiaArmsBusulfan, Fludarabine, Total-body irradiation, Cyclophosphamide, Melphalan
3 · Its place in the literature

Who cites it

18 citing papers in PubMed, 2 syntheses or guidelines pooled it.

  1. Guideline
  2. Guideline
  3. Article
  4. Article
  5. Article
  6. Article
  7. Article
  8. Article
  9. Review
  10. Observational
  11. Article
  12. Article
  13. Article
  14. Review
  15. Article
  16. Donor selection, graft engineering, and dosing oh my!Hematology. American Society of Hematology. Education Program · 2025
    Review
  17. Review
  18. Review
4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

33 authors.

Monzr M Al MalkiCity of Hope National Medical Center, Duarte, CA.ORCID 0000-0001-8226-471X
Stephanie Bo-SubaitCIBMTR (Center for International Blood and Marrow Transplant Research), NMDP, Minneapolis, MN.
Brent LoganDivision of Biostatistics, Data Science Institute, Medical College of Wisconsin, Milwaukee, WI.ORCID 0000-0002-4473-4044
Janelle OlsonCIBMTR (Center for International Blood and Marrow Transplant Research), NMDP, Minneapolis, MN.
Jianqun KouCIBMTR (Center for International Blood and Marrow Transplant Research), NMDP, Minneapolis, MN.
Sarah SmithCIBMTR (Center for International Blood and Marrow Transplant Research), NMDP, Minneapolis, MN.
Erin LeckroneCIBMTR (Center for International Blood and Marrow Transplant Research), NMDP, Minneapolis, MN.
Juan WuCIBMTR (Center for International Blood and Marrow Transplant Research), NMDP, Minneapolis, MN.
Heather E StefanskiCIBMTR (Center for International Blood and Marrow Transplant Research), NMDP, Minneapolis, MN.
Jeffery J AulettaCIBMTR (Center for International Blood and Marrow Transplant Research), NMDP, Minneapolis, MN.ORCID 0000-0002-1515-2141
Stephen R SpellmanCIBMTR (Center for International Blood and Marrow Transplant Research), NMDP, Minneapolis, MN.ORCID 0000-0002-7271-8252
Craig MalmbergCIBMTR (Center for International Blood and Marrow Transplant Research), NMDP, Minneapolis, MN.
Medhat AskarCIBMTR (Center for International Blood and Marrow Transplant Research), NMDP, Minneapolis, MN.ORCID 0000-0003-2633-9363
Rachel CusatisCIBMTR (Center for International Blood and Marrow Transplant Research), Medical College of Wisconsin, Milwaukee, WI.ORCID 0000-0001-6252-6516
Brian C ShafferAdult Bone Marrow Transplantation Service, Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, NY.ORCID 0000-0003-3433-9179
Dipenkumar ModiBarbara Ann Karmanos Cancer Institute, Wayne State University, Detroit, MI.ORCID 0000-0001-6525-8844
Farhad KhimaniDepartment of Blood and Marrow Transplantation and Cellular Immunotherapy, Moffitt Cancer Center, Tampa, FL.ORCID 0000-0003-4828-4711
Mahasweta GooptuDepartment of Hematology/Oncology, Dana-Farber Cancer Institute, Boston, MA.
Mehdi HamadaniCIBMTR (Center for International Blood and Marrow Transplant Research), Medical College of Wisconsin, Milwaukee, WI.ORCID 0000-0001-5372-510X
Abeer MadboulyCIBMTR (Center for International Blood and Marrow Transplant Research), NMDP, Minneapolis, MN.ORCID 0000-0003-4199-3435
Martin MaiersCIBMTR (Center for International Blood and Marrow Transplant Research), NMDP, Minneapolis, MN.ORCID 0000-0002-0198-2064
Stephanie FingersonCIBMTR (Center for International Blood and Marrow Transplant Research), NMDP, Minneapolis, MN.ORCID 0000-0003-1960-9695
Rachel CookOregon Health & Science University, Portland, OR.
Karen BallenDivision of Hematology/Oncology, University of Virginia Health, Charlottesville, VA.ORCID 0000-0003-1706-3975
Alison LorenDivision of Hematology/Oncology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA.ORCID 0000-0002-8812-9911
Karilyn LarkinThe Ohio State University Comprehensive Cancer Center, Columbus, OH.ORCID 0000-0002-1898-9836
Sally AraiStanford Health Care, Palo Alto, CA.
Muna QayedAflac Cancer and Blood Disorders Center, Emory University and Children's Healthcare of Atlanta, Atlanta, GA.ORCID 0000-0001-7689-343X
Sung Won ChoiUniversity of Michigan, Blood and Marrow Transplantation Program, Ann Arbor, MI.ORCID 0000-0002-6321-3834
Larisa BroglieCIBMTR (Center for International Blood and Marrow Transplant Research), Medical College of Wisconsin, Milwaukee, WI.ORCID 0000-0002-1674-048X
Bronwen E ShawCIBMTR (Center for International Blood and Marrow Transplant Research), Medical College of Wisconsin, Milwaukee, WI.ORCID 0000-0003-3317-1896
Steven Michael DevineCIBMTR (Center for International Blood and Marrow Transplant Research), NMDP, Minneapolis, MN.ORCID 0000-0001-7731-759X
Antonio Martin Jimenez JimenezDivision of Transplantation and Cellular Therapy, University of Miami Miller School of Medicine, Miami, FL.ORCID 0000-0003-2817-6836

Funding

X-RAY CRYSTALLOGRAPHYP30CA008748 · NCI · SLOAN-KETTERING INSTITUTE FOR CANCER RES · PI SELWYN M VICKERS · 1985 to 2026
$347.4M
Translational Therapeutics Research Program (TT)P30CA016058 · NCI · OHIO STATE UNIVERSITY · PI Daniel G. Stover · 1985 to 2026
$132.3M
Data Resource for Analyzing Blood &Marrow TransplantsU24CA076518 · NCI · MEDICAL COLLEGE OF WISCONSIN · PI Amy M Moskop, Bronwen Shaw · 1998 to 2026
$105.2M
Blood and Marrow Transplant Clinical Trials Network DCC- The Medical College of Wisconsin, Inc.U24HL138660 · NHLBI · MEDICAL COLLEGE OF WISCONSIN · PI Steven M. DeVine, Mehdi Hussain Hamadani · 2017 to 2026
$71.4M
NTP INFORMATION SYSTEMS SUPPORT27305C0011 · NIEHS · Z-TECH CORPORATION · 2007 to 2009
$4.3M
Clinical and mechanistic studies defining optimal preparative approaches to infants with IL2RG/JAK3/RAG1/RAG2 SCID: a randomized trial of busulfan dosageU01AI184132 · NIAID · NATIONAL MARROW DONOR PROGRAM · PI JEFFERY J AULETTA, Michael A Pulsipher · 2024 to 2026
$3.2M
BMT CTN Core - Dana-Farber Cancer Institute and MGH ConsortiumUG1HL069249 · NHLBI · DANA-FARBER CANCER INST · PI Mahasweta Gooptu · 2017 to 2026
$2.2M
BMT Core - Pediatric Transplantation and Cellular Therapy Consortium (PTCTC): Providing Clinical Trial Access For Children With Life Threatening DiseasesUG1HL174426 · NHLBI · NATIONAL MARROW DONOR PROGRAM · PI Leslie S Kean, Heather E Stefanski · 2024 to 2026
$513k
PROVIDE RABBITS, RATS, MICE, HAMSTERS, GERBILS, GUINEA PIGS27307C0011 · NIEHS · PI BOLEN, WAYNE · 2007 to 2007
$500k
NCI NIH HHS P30 CA008748NCI NIH HHS P30 CA016058NCI NIH HHS U24 CA076518NHLBI NIH HHS U24 HL138660NHLBI NIH HHS UG1 HL069249NHLBI NIH HHS UG1 HL174426NIAID NIH HHS U01 AI184132NIEHS NIH HHS 27305C0011NIEHS NIH HHS 27307C0011NIEHS NIH HHS 27398C0011
6 · The paper itself

Abstract

purposeAllogeneic hematopoietic stem cell transplantation (HSCT) is a curative treatment for advanced hematologic malignancies. HSCT using human leukocyte antigen (HLA)-mismatched donors is historically associated with inferior survival. Patients from underrepresented racial and ethnic groups more frequently rely on HLA-mismatched donors. We hypothesized that post-transplant cyclophosphamide (PTCy) based graft versus host disease (GVHD) prophylaxis would improve outcomes for HSCT recipients using peripheral blood stem cells (PBSCs) from HLA-mismatched unrelated donors (MMUDs) by reducing the risk of GVHD.

methodsThis phase II, nonrandomized, multicenter trial assessed PBSCs in the setting of a GVHD prophylaxis regimen of cyclophosphamide, tacrolimus, and mycophenolate mofetil in two adult strata: myeloablative conditioning (MAC) and reduced-intensity or nonmyeloablative (RIC/NMA) conditioning before HSCT from a MMUD. The primary objective was to estimate 1 year overall survival (OS) for each stratum. Key secondary end points included incidences of acute and chronic GVHD.

resultsA total of 145 patients enrolled, with 59% self-identifying within an underrepresented group. The 1 year OS was 83.8% (95% CI, 73.1% to 90.4%) for MAC and 78.6% (95% CI, 67% to 86.5%) for RIC/NMA. Incidences of grades III to IV acute GVHD at 6 months were 8% (95% CI, 3.2 to 15.6) for MAC and 10% (95% CI, 4.4 to 18.4) for RIC/NMA. Moderate/severe chronic GVHD at 1 year was 10.3% (95% CI, 4.4 to 18.9) for MAC and 8.6% (95% CI, 3.5 to 16.6) for RIC/NMA. 32% of patients whose donors matched at fewer than seven of eight HLA alleles had similar OS compared with those with donor matched at seven of eight alleles.

conclusionPTCy-based GVHD prophylaxis after MMUD HSCT with PBSC grafts results in favorable 1 year OS. Using MMUDs expands donor availability to all patients regardless of ancestry (ACCESS; ClinicalTrials.gov identifier: NCT04904588).

Indexed as

CyclophosphamideGraft vs Host DiseaseHematologic NeoplasmsImmunosuppressive AgentsPeripheral Blood Stem Cell TransplantationUnrelated DonorsAdolescentAdultAgedFemaleHLA AntigensHumansMaleMiddle AgedMycophenolic AcidTacrolimusCyclophosphamideHLA AntigensImmunosuppressive AgentsMycophenolic AcidTacrolimus

Identifiers

PMID40523209
PMCPMC12353616

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
Read underepoch 390

Registered trials

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.