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Clinical Trial
. 2025 Mar 10;43(8):912-918.
doi: 10.1200/JCO.24.00921. Epub 2025 Jan 3.

Improved Patient-Reported Outcomes With Post-Transplant Cyclophosphamide: A Quality-of-Life Evaluation and 2-Year Outcomes of BMT CTN 1703

Shernan G Holtan  1 , Javier Bolaños-Meade  2 , Monzr M Al Malki  3 , Juan Wu  4 , Carrie L Kitko  5 , Ran Reshef  6 , Andrew R Rezvani  7 , Brian C Shaffer  8   9 , Melhem M Solh  10 , Janny M Yao  11 , Lyndsey Runaas  12 , Hany Elmariah  13 , Karilyn T Larkin  14 , Najla El Jurdi  15 , Mahasweta Gooptu  16 , Alison W Loren  17 , Aric C Hall  18 , Amin M Alousi  19 , Omer Jamy  20 , William Clark  21 , Leslie Kean  22 , Ami S Bhatt  23 , Miguel-Angel Perales  8   9 , Kristy Applegate  4 , Yvonne Adeduni Efebera  24 , Eric Leifer  25 , Richard J Jones  2 , Mary M Horowitz  26   27 , Deborah Mattila  28 , Wael Saber  27 , Mehdi Hamadani  27 , Michael J Martens  26   29
Affiliations
Clinical Trial

Improved Patient-Reported Outcomes With Post-Transplant Cyclophosphamide: A Quality-of-Life Evaluation and 2-Year Outcomes of BMT CTN 1703

Shernan G Holtan et al. J Clin Oncol. .

Abstract

The BMT CTN 1703 phase III trial confirmed that graft-versus-host disease (GVHD) prophylaxis with post-transplantation cyclophosphamide (PTCy), tacrolimus (Tac), and mycophenolate mofetil (MMF) results in superior GVHD-free, relapse-free survival (GRFS) compared with Tac/methotrexate (MTX) prophylaxis. This companion study assesses the effect of these regimens on patient-reported outcomes (PROs). Using the Lee Chronic GVHD Symptom Score and PROMIS subscales (physical function, GI symptoms, social role satisfaction) as primary end points and hemorrhagic cystitis symptoms and Lee subscales as secondary end points, responses from English and Spanish speakers were analyzed at baseline and days 100, 180, and 365 after transplant. PRO scores were compared between the arms using inverse probability weighted-independent estimating equation models. The PTCy arm had significantly lower scores on the Lee Chronic GVHD Symptom Scale (P = .01), indicating lower GVHD symptom burden. Lee Scale nutrition and mouth subscores were also better in the PTCy arm compared with the Tac/MTX arm (P < .01 for both). Older participants (age >65 years) reported better Lee Scale psychological subscores than younger participants (P = .003). No significant differences were identified in hemorrhagic cystitis or in the PROMIS subscales between treatment arms. The updated clinical end points at 2 years for the parent trial confirmed that PTCy/Tac/MMF maintained a significant advantage over Tac/MTX in GRFS (42.4% v 28.8%, P = .001). In addition to improved GRFS, patients randomly assigned to the PTCy arm reported lower symptom burden during the first year after transplant.

Trial registration: ClinicalTrials.gov NCT03959241.

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Conflict of interest statement

AUTHORS’ DISCLOSURES OF POTENTIAL CONFLICTS OF INTEREST

Disclosures provided by the authors are available with this article at DOI https://doi.org/10.1200/JCO.24.00921.

AUTHORS’ DISCLOSURES OF POTENTIAL CONFLICTS OF INTEREST

The following represents disclosure information provided by authors of this manuscript. All relationships are considered compensated unless otherwise noted. Relationships are self-held unless noted. I = Immediate Family Member, Inst = My Institution. Relationships may not relate to the subject matter of this manuscript. For more information about ASCO’s conflict of interest policy, please refer to www.asco.org/rwc or ascopubs.org/jco/authors/author-center.

Open Payments is a public database containing information reported by companies about payments made to US-licensed physicians (Open Payments).

Shernan G. Holtan

Consulting or Advisory Role: Incyte, Bristol Myers Squibb, CSL Behring (Inst), Sanofi, MaaT Pharma

Research Funding: Incyte, VITRAC Therapeutics

Open Payments Link: https://openpaymentsdata.cms.gov/physician/471226

Javier Bolaños-Meade

Honoraria: Banner MD Anderson Colorado

Consulting or Advisory Role: MJH Healthcare Holdings LLC, Avoro Capital Advisors

Travel, Accommodations, Expenses: Dictaforum Servicios

Monzr M. Al Malki

Consulting or Advisory Role: CareDX, T Scan, TR1X

Research Funding: NexImmune, Incyte, Stemline Therapeutics

Carrie L. Kitko

Consulting or Advisory Role: Incyte, Sanofi

Travel, Accommodations, Expenses: Adaptive Biotechnologies

Ran Reshef

Consulting or Advisory Role: Gilead Sciences, TScan Therapeutics, Incyte, Orca Bio, Allogene Therapeutics, Quell Biotherapeutics, Sana Biotechnology, Autolus Therapeutics

Research Funding: Atara Biotherapeutics (Inst), Incyte (Inst), Immatics (Inst), Takeda (Inst), Gilead Sciences (Inst), Precision Biosciences (Inst), Bristol Myers Squibb (Inst), Janssen (Inst), Sanofi (Inst), TCR2 Therapeutics (Inst), CareDX (Inst), Synthekine (Inst), AbbVie (Inst), TScan Therapeutics (Inst), Genentech/Roche (Inst), Cabaletta Bio (Inst)

Expert Testimony: Bayer

Travel, Accommodations, Expenses: Gilead Sciences, Janssen

Andrew R. Rezvani

Research Funding: Gamida Cell (Inst)

Open Payments Link: https://openpaymentsdata.cms.gov/physician/750939

Brian C. Shaffer

Consulting or Advisory Role: Hansa Biopharma

Melhem M. Solh

Speakers’ Bureau: Bristol Myers Squibb, Amgen, Seagen, GlaxoSmithKline

Research Funding: Partner therapeutics

Lyndsey Runaas

Consulting or Advisory Role: Agios, Blueprint Medicines

Hany Elmariah

Consulting or Advisory Role: Shoreline Biosciences

Research Funding: Bristol Myers Squibb/Pfizer

Karilyn T. Larkin

Research Funding: Debiopharm Group

Uncompensated Relationships: Debiopharm Group

Mahasweta Gooptu

Consulting or Advisory Role: Syndax

Travel, Accommodations, Expenses: Syndax

Alison W. Loren

This author is a member of the Journal of Clinical Oncology Editorial Board. Journal policy recused the author from having any role in the peer review of this manuscript.

Research Funding: Equillium (Inst)

Amin M. Alousi

Honoraria: Generon, Genentech, Kadmon, Prolacta Bioscience

Consulting or Advisory Role: Incyte, Sanofi

Omer Jamy

Consulting or Advisory Role: Ascentage Pharma, Terns Pharmaceuticals, MaaT Pharma

William Clark

Employment: Virginia Commonwealth University

Consulting or Advisory Role: Caribou Biosciences

Research Funding: AbbVie (Inst), Caribou Biosciences (Inst), GPCR Therapeutics (Inst), ImmPACT-Bio (Inst), Janssen (Inst), Kite/Gilead (Inst)

Leslie Kean

Consulting or Advisory Role: HiFiBiO Therapeutics, Vertex

Research Funding: Regeneron, Bristol Myers Squibb, Tonix, Tessera

Patents, Royalties, Other Intellectual Property: Licensing Fees for ABA2 clinical trial data

Ami S. Bhatt

Employment: January AI (I), Varian Medical Systems (I)

Leadership: Stylus Medicine

Stock and Other Ownership Interests: Caribou Biosciences

Consulting or Advisory Role: Arc Bio, January AI (I), Kaleido Biosciences, Caribou Biosciences, BiomX

Patents, Royalties, Other Intellectual Property: I have a single patent pending related to SARS-CoV-2 detection in fecal samples, My spouse has several patents pending related to the use of artificial intelligence for blood glucose prediction and radiation therapy planning (I)

Travel, Accommodations, Expenses: Stylus Medicine

Miguel-Angel Perales

Stock and Other Ownership Interests: NexImmune, Orca Bio, Omeros

Consulting or Advisory Role: Incyte, Merck, NexImmune, Novartis, Medigene, Nektar, Cidara Therapeutics, Celgene, Kite/Gilead, Bristol Myers Squibb, Omeros, Vor Biopharma, Adicet Bio, Allogene Therapeutics, AlloVir, Caribou Biosciences, Equillium, Exivir, Sanofi

Research Funding: Incyte (Inst), Miltenyi Biotec (Inst), Novartis (Inst) Kite, a Gilead company (Inst), Nektar (Inst)

Yvonne Adeduni Efebera

Honoraria: Takeda, Oncopeptides, Alnylam, Sanofi, GlaxoSmithKline, ORCA Therapeutics

Consulting or Advisory Role: Oncopeptides, Sanofi, Janssen Sanofi, Pfizer

Speakers’ Bureau: Takeda, Janssen, Akcea Therapeutics

Research Funding: Bristol Myers Squibb/Celgene

Mary M. Horowitz

Consulting or Advisory Role: Medac (Inst)

Research Funding: Novartis (Inst), Sanofi (Inst), Astellas Pharma (Inst), Incyte (Inst)

Mehdi Hamadani

Consulting or Advisory Role: ADC Therapeutics, Puma Biotechnology (I), Kite/Gilead, Omeros, Seagen, Genmab, Myeloid Therapeutics, BeiGene, AstraZeneca, Sanofi, Bristol Myers Squibb/Celgene, crispr therapeutics, Caribou Biosciences, AbbVie, Genentech, Forte Biosciences

Speakers’ Bureau: Genzyme, AstraZeneca, BeiGene, ADC Therapeutics, Kite/Gilead

Research Funding: Takeda, Spectrum Pharmaceuticals, Otsuka, Astellas Pharma, Genzyme, Genzyme

No other potential conflicts of interest were reported.

Figures

FIG 1.
FIG 1.
CONSORT diagram. ITT, intention to treat; MMF, mycophenolate mofetil; MTX, methotrexate; PTCy, post-transplantation cyclophosphamide; QOL, quality of life; Tac, tacrolimus.
FIG 2.
FIG 2.
Box and scatter plots for (A) Lee Chronic GVHD Symptom Scale and (B-D) PROMIS subscales by treatment arm. GVHD, graft-versus-host disease; MTX, methotrexate; PTCy, post-transplantation cyclophosphamide; Tac, tacrolimus; TX, transplant.
FIG 3.
FIG 3.
IPW-IEE model estimated mean scores for (A) Lee Chronic GVHD Symptom Scale and (B-D) PROMIS subscales by treatment arm. Higher score indicates greater burden of symptoms. GVHD, graft-versus-host disease; IPW-IEE, inverse probability weighted-independent estimating equation; MTX, methotrexate; PTCy, post-transplantation cyclophosphamide; Tac, tacrolimus; TX, transplant.
See this image and copyright information in PMC

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