Results from the prospective phase 2 multicentre UK PBSC haplo trial using PTCy pre- or post-stem cells
deLavallade, Hugues Wilson, William Protheroe, Rachel Smith, Matthew Edwards, Darren O'Donnell, Katie Tholouli, Eleni Paneesha, Shankaranarayan Collin, Matthew Uttenthal, Ben
deLavallade, Hugues
Wilson, William
Protheroe, Rachel
Smith, Matthew
Edwards, Darren
O'Donnell, Katie
Tholouli, Eleni
Paneesha, Shankaranarayan
Collin, Matthew
Uttenthal, Ben
Abstract
This prospective phase 2 multicentre non-randomised parallel arm study of haploidentical peripheral blood stem cell (PBSC) transplantation with post-transplant cyclophosphamide (PTCy) recruited 77 patients: 50 received reduced-intensity conditioning (RIC) with PTCy post stem cell infusion while 27 underwent myeloablative conditioning (MAC) with PTCy given after lymphocyte but prior to infusion of CD34 selected stem cells. The primary end-point 1-year overall survival (OS) was 86% for RIC and 78% for MAC, meeting the pre-specified targets for efficacy. At 4 years, OS was 63% for RIC and 60% for MAC with low non-relapse mortality of RIC 18% and MAC 4%. Engraftment was faster in the MAC arm (median time to neutrophil and platelet engraftment 11 and 15 days; 21 and 24 days in the RIC arm). The economic analysis showed that MAC had higher initial transplant costs, RIC incurred greater post-transplant monitoring costs, resulting in higher overall costs (RIC £156 711, MAC £131 092). Quality of life (QoL) outcomes in both indicated significant post-transplant declines at 3 months but returned to baseline by 12 months. This study confirms the long-term safety of using PBSC with PTCy in haploidentical transplants, with a good quality of life and reasonable costs.
MIDER Authors
Affiliations
Guys and St Thomas' NHS Foundation Trust; Kings College Hospital NHS Foundation Trust; King's College London; University College London; University Hospital Bristol NHS Foundation Trust; Barts Health NHS Trust; Manchester University NHS Foundation Trust; University Hospitals Birmingham NHS Foundation Trust; Newcastle Upon Tyne Hospitals NHS Foundation Trust; Cambridge University Hospital NHS Foundation Trust; NHS Greater Glasgow and Clyde; University of Sheffield; University College London NHS Foundation Trust; Sheffield Teaching Hospitals NHS Foundation Trust
Date
2026-03-25
Type
Article
Collections
Citation
deLavallade H, Wilson W, Protheroe R, Smith M, Edwards D, O'Donnell K, Tholouli E, Paneesha S, Collin M, Uttenthal B, Irvine D, Yee M, Tappenden P, Kottaridis P, Snowden JA, Mackinnon S, Pagliuca A, Raj K. Results from the prospective phase 2 multicentre UK PBSC haplo trial using PTCy pre- or post-stem cells. Br J Haematol. 2026 May;208(5):1758-1767. doi: 10.1111/bjh.70429. Epub 2026 Mar 25.