Chronic graft-versus-host disease in the era of posttransplant cyclophosphamide
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Autores de IIS La Fe
Participantes ajenos a IIS La Fe
- León G.
- Vallejo N.
Grupos
Abstract
Chronic graft-versus-host disease (cGVHD) remains a leading cause of late morbidity after allogeneic hematopoietic cell transplantation (HCT), but its phenotype under modern prophylaxis with post-transplant cyclophosphamide (PTCy) is not well characterized. We conducted a prospective, single-center study of 600 consecutive adults undergoing HCT with PT-Cy-based prophylaxis to assess incidence, clinical manifestations, treatment response, prognostic factors, and outcomes. Donors included matched siblings (36%), matched unrelated (34%), haploidentical (24%), and mismatched unrelated (6%). The 1-year cumulative incidence of moderate-to-severe cGVHD was 22% (95% confidence interval [CI]: 19-26%). The mouth was the most frequently involved organ (64%), with lichen planus-like changes as the predominant diagnostic feature, whereas sclerotic forms were uncommon. Notably, 27% of moderate-to-severe cases were managed successfully without systemic corticosteroids. The cumulative incidence of systemic therapy requirement was 15% at 1 year, with risk significantly higher in donors >30 years and in female-to-male transplants. Among 105 patients requiring systemic steroids, 64% achieved complete response, 32% discontinued immunosuppression, yet 18% developed cGVHD-related sequelae. Mouth ulcers and erythema, as well as a lung score >2 at steroid initiation independently predicted shorter failure-free survival. At 2 years, overall survival, cGVHD-free relapse-free survival, and GVHD-free relapse-free survival were 76% (95% CI: 72-79), 63% (95% CI: 60-68), and 57% (95% CI: 53-62), respectively. In conclusion, after HCT with PTCy-based prophylaxis, systemic therapy was required in only a minority of patients, with risk influenced by donor age and sex mismatch rather than donor type. While corticosteroids were generally effective, a substantial subset required salvage therapy, underscoring the burden of refractory cGVHD and the need for steroid-sparing approaches and novel interventions. © 2026 Ferrata Storti Foundation Published under a CC BY-NC license
Datos de la publicación
- ISSN/ISSNe:
- 0390-6078, 1592-8721
- Tipo:
- Article
- Páginas:
- 2366-2376
- PubMed:
- 41568541
- Enlace a otro recurso:
- www.scopus.com
- Factor de Impacto:
- 2,528 SCImago ℠
- Cuartil:
- Q1 SCImago ℠
HAEMATOLOGICA FERRATA STORTI FOUNDATION
Citas Recibidas en Web of Science: 5
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- No hay documentos
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Keywords
- Adolescent; Adult; Aged; Chronic Disease; Cyclophosphamide; Female; Graft vs Host Disease; Hematopoietic Stem Cell Transplantation; Humans; Immunosuppressive Agents; Incidence; Male; Middle Aged; Prospective Studies; Transplantation, Homologous; Treatment Outcome; Young Adult; belumosudil; cyclophosphamide; inotuzumab ozogamicin; mycophenolate mofetil; posttransplant cyclophosphamide; prednisone; rituximab; ruxolitinib; sirolimus; tacrolimus; unclassified drug; cyclophosphamide; immunosuppressive agent; acute leukemia; adult; aged; allogeneic hematopoietic cell transplantation; alopecia; anemia; Article; autoimmune thrombocytopenia; cardiomyopathy; cell transplantation; chronic graft versus host disease; computer assisted tomography; disease activity; dystrophy; erythema; failure free survival; fasciitis; female; hair loss; hematolytic anemia; human; hypergammaglobulinemia; hyperpigmentation; immunosuppressive treatment; lung transplantation; lymphoma; major clinical study; male; match
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