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Optimal dosing of MMF with PTCy in patients undergoing haploidentical PBSCT
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A retrospective analysis assessed the impact of a lower relative dose of MMF on outcomes in adult patients undergoing haplo-allo-HSCT with PTCy. This analysis included 386 patients undergoing haplo-PBSTC at City of Hope National Medical Center or Moffit Cancer Center from April 2014 to August 2020. Patients were categorized as receiving a low (<29 mg/kg/day; 14%), low-intermediate (29–34 mg/kg/day; 19%), high-intermediate (35–41 mg/kg/day; 35%), or high (>41 mg/kg/day; 32%) MMF dose. Results from this analysis were published in Transplantation and Cellular Therapy by Elmariah et al.1 |
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The 2-year probability of relapse was 38%, 26%, 28%, and 21% for the high, high-intermediate, low-intermediate, and low dose groups, respectively (p = 0.12), and the 2-year probability of PFS was 48%, 57%, 57%, and 70%, respectively (p = 0.04). Multivariable analysis demonstrated that low MMF dose exposure was associated with a lower risk of relapse (HR, 0.45; 95% CI, 0.21–0.94; p = 0.03) and improved PFS (HR, 0.58; 95% CI, 0.34–0.99; p = 0.045) vs the high dose exposure. |
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MMF exposure did not impact engraftment rates, acute or chronic GvHD rates, or NRM, indicating that lower MMF doses do not compromise these outcomes. |
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The 2-year estimated OS rates were 58%, 64%, 65%, and 80% for the high, high-intermediate, low-intermediate, and low dose groups, respectively (p = 0.04). However, multivariable analysis demonstrated that the dose of MMF did not contribute to OS. |
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These findings suggest that a lower dose of MMF could improve outcomes in patients undergoing haplo-PBSCT with PTCy, warranting future prospective studies to identify the optimal MMF dosing strategy in this setting. |
Abbreviations: allo-HSCT, allogeneic hematopoietic stem cell transplantation; CI, confidence interval; GvHD, graft-versus-host disease; haplo, haploidentical; HR, hazard ratio; MMF, mycophenolate mofetil; NRM, non-relapse mortality; OS, overall survival; PBSCT, peripheral blood stem cell transplantation; PFS, progression-free survival; PTCy, post-transplant cyclophosphamide.
- Elmariah H, Otoukesh S, Kumar A, et al. Lower weight-based mycophenolate mofetil dosing is associated with superior outcomes after haploidentical hematopoietic cell transplant with post-transplant cyclophosphamide. Transplant Cell Ther. Online ahead of print. DOI: 10.1016/j.jtct.2024.07.024
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