Introduction: Graft versus host disease [GVHD] remains the major factor underlying transplant related morbidity and mortality among recipients of allogeneic stem cell transplant [allo-SCT]. The choice of GVHD prophylaxis is largely determined by HLA disparity between the donor and the recipient and the intensity of the conditioning regimen. Mycophenolate mofetil [MMF] plus calcineurin inhibitor [CNI] combination is mostly used in reduced intensity [RI] and non-myeloablative conditioning [NMAC] whereas methotrexate and CNI combination is preferred in myeloablative conditioning [MAC]. We present single institution outcomes in patients undergoing allo-SCT with MMF and CNI as the only combination for GVHD prophylaxis.

Methods: Data from all adult patients who underwent allo-SCT at our institution from 2007 to 2016 was collected from our Center for Blood and Marrow Transplant Research [CIBMTR] portal. Only patients who received both MMF and CNI as GVHD prophylaxis were included in the analysis.

Results: Total 141 patients [table 1] were included with the median age of 50 years. Most patients were female [52%] and white [85%]. Disease characteristics included AML 56.7%, ALL 14.2%, CML 7.7%, MDS/MPD 7.7%, HL/NHL 6.4% and others 7%. MAC was used in 75.2% whereas NMAC/RI conditioning was used in 24.8% of the patients. Peripheral blood was the predominant source of stem cell graft [81%]. Donor status included 30.5% HLA matched related, 63.8% HLA mismatched/matched unrelated and 4.9% HLA mismatch related. Incidence of acute GVHD at day 100 was 49.6% [grade I 16.5%; grade II-IV 33.1%], acute GVHD at day 365 was 59.6%, and chronic GVHD at day 365 was 48.1% [limited 13.5%; extensive 34.6%]. One year overall survival [OS] was 51% [95% CI 42-59%]. OS was significantly associated with race (p = 0.033) and conditioning regimen (p = 0.036), favoring better survival with white race and NMAC/RIC regimen.

Discussion: Based on historical cohort, combination of methotrexate and tacrolimus is associated with acute graft versus host disease in 89% [grade I 11%; grade II-IV 78%], chronic graft versus host disease in 94% [limited 32%; extensive 65%] and one year overall survival of 47%. Hence our single institutional analysis shows that the combination of mycophenolate and tacrolimus is superior to methotrexate and tacrolimus combination for prevention of acute and chronic graft versus host disease as well as 1-year overall survival. We suggest that well-designed randomized controlled clinical trials be conducted to verify these findings.

Disclosures

No relevant conflicts of interest to declare.

Author notes

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Asterisk with author names denotes non-ASH members.

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