JCO oncology practice · 2026
Impact of Graft-Versus-Host Disease Prophylaxis on Long-Term Outcomes in Patients Surviving Beyond Two Years After Allogeneic Transplantation.
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Desai Nihar, Rodriguez-Rodriguez Sergio, Chen Carol, Al-Shaibani Eshrak, Alfaro Moya Tommy, Novitzky-Basso Igor, Pasic Ivan, Michelis Fotios V, Viswabandya Auro, Kim Dennis D H, Kumar Rajat, Mattsson Jonas, Law Arjun D
Аннотация
PURPOSE: Post-transplant cyclophosphamide (PTCy) is increasingly used for graft-versus-host disease (GVHD) prophylaxis, but long-term outcomes remain incompletely defined.
PATIENTS AND METHODS: We analyzed 874 relapse-free survivors at 2 years from 2,046 patients who underwent allogeneic hematopoietic stem-cell transplantation (HSCT) for hematologic malignancies between 2010 and 2022. GVHD prophylaxis included PTCy plus rabbit antithymocyte globulin (ATG) and a calcineurin inhibitor (CnI; Group I, n = 429) or alternative regimens (Group II, n = 445).
RESULTS: With an overall follow-up of 5,275 person-years after HSCT, overall survival (OS) 3 years after the landmark was 88.7% (95% CI, 86 to 91). Patients receiving ATG-PTCy-CnI had improved outcomes compared with those receiving alternative regimens, with OS of 93.2% versus 85.0% (P < .001), and lower nonrelapse mortality (NRM; 3.4% v 10.8%; P < .001). On multivariable analysis, ATG-PTCy-CnI was independently associated with improved OS (hazard ratio [HR], 0.41; 95% CI, 0.21 to 0.79; P = .007) and lower NRM (HR, 0.34; 95% CI, 0.17 to 0.74; P = .006). Chronic GVHD remained the leading cause of late mortality; deaths from GVHD, infections, treatment-related toxicity, and second primary malignancies were all reduced with ATG-PTCy-CnI. The cumulative incidence of relapse was similar between groups. Standardized mortality ratios were 1.32 for ATG-PTCy-CnI and 2.06 for other regimens, corresponding to a 1.6-fold higher excess mortality with alternative prophylaxis (P = .036).
CONCLUSION: Among patients alive and relapse-free at least 2 years after HSCT, long-term outcomes were excellent. The use of ATG-PTCy-CnI prophylaxis was associated with further improvements in survival through reductions in GVHD-related mortality and late complications.
PATIENTS AND METHODS: We analyzed 874 relapse-free survivors at 2 years from 2,046 patients who underwent allogeneic hematopoietic stem-cell transplantation (HSCT) for hematologic malignancies between 2010 and 2022. GVHD prophylaxis included PTCy plus rabbit antithymocyte globulin (ATG) and a calcineurin inhibitor (CnI; Group I, n = 429) or alternative regimens (Group II, n = 445).
RESULTS: With an overall follow-up of 5,275 person-years after HSCT, overall survival (OS) 3 years after the landmark was 88.7% (95% CI, 86 to 91). Patients receiving ATG-PTCy-CnI had improved outcomes compared with those receiving alternative regimens, with OS of 93.2% versus 85.0% (P < .001), and lower nonrelapse mortality (NRM; 3.4% v 10.8%; P < .001). On multivariable analysis, ATG-PTCy-CnI was independently associated with improved OS (hazard ratio [HR], 0.41; 95% CI, 0.21 to 0.79; P = .007) and lower NRM (HR, 0.34; 95% CI, 0.17 to 0.74; P = .006). Chronic GVHD remained the leading cause of late mortality; deaths from GVHD, infections, treatment-related toxicity, and second primary malignancies were all reduced with ATG-PTCy-CnI. The cumulative incidence of relapse was similar between groups. Standardized mortality ratios were 1.32 for ATG-PTCy-CnI and 2.06 for other regimens, corresponding to a 1.6-fold higher excess mortality with alternative prophylaxis (P = .036).
CONCLUSION: Among patients alive and relapse-free at least 2 years after HSCT, long-term outcomes were excellent. The use of ATG-PTCy-CnI prophylaxis was associated with further improvements in survival through reductions in GVHD-related mortality and late complications.