SCT/BMT clinical trials
22 SCT/BMT trials (Malignant Hematology) summarized for fellows and board review, newest first within each disease. Each summary covers design, results, toxicity and what changed in practice.
HSC Transplant (22)
- Haplo GVHD prophylaxis: low-dose ATG/PTCy vs ATG vs PTCy Blood 2026The three prophylaxis strategies yielded similar grade 2-4 aGVHD and survival after haplo-PBSCT. Low-dose ATG/PTCy delivered faster hematologic recovery with...
- Low-dose ruxolitinib GVHD prophylaxis in haploidentical HSCT Lancet Haematol 2026Substituting low-dose ruxolitinib for mycophenolate mofetil within an ATG, calcineurin inhibitor, and short-course methotrexate backbone reduced grade II-IV acute...
- PRECISION-T (Orca-T) Blood 2026Orca-T met its primary endpoint of improved moderate-to-severe cGVHD-free survival versus standard Tac/MTX prophylaxis with conventional allografts in MAC HLA-matched...
- PTCy-Ruxolitinib vs PTCy-Tac-MMF (phase 2 noninferiority) Transplant Cell Ther 2026PTCy-Ruxo was noninferior to standard PTCy-Tac-MMF for prevention of grade II-IV acute GVHD at day +125 in unrelated and haploidentical donor alloHCT, with a...
- ALLG BM12 CAST NEJM 2025Adding post-transplant cyclophosphamide to a calcineurin inhibitor (replacing methotrexate) produced significantly longer GVHD-free, relapse-free survival than...
- Itacitinib + PTCy (haploidentical HCT) Blood 2025Adding itacitinib to PTCy-based prophylaxis in haploidentical HCT produced very low rates of CRS, acute and chronic GVHD, and NRM, with encouraging GRFS and overall...
- Ruxolitinib maintenance after allo-HCT Blood 2025Prolonged ruxolitinib maintenance after reduced-intensity allo-HCT was associated with low rates of clinically significant chronic GVHD and favorable GRFS....
- AGAVE-201 NEJM 2024Axatilimab 0.3mg/kg q2w demonstrated a 74% ORR in SR-cGVHD including fibrotic manifestations, supporting FDA approval as the first anti-CSF1R and first...
- REACH4 Blood 2024Ruxolitinib demonstrated a 75.6% ORR at day 28 in pediatric SR-aGVHD, consistent with adult REACH2 data, establishing ruxolitinib with a weight-based regimen as a...
- BMT CTN 1703 NEJM 2023PTCy-based prophylaxis significantly improved GRFS vs both Tac/MTX and CD34+ selection in HLA-matched donor PBSC allo-HCT, driven by substantially lower acute and...
- HARMONY Lancet Haematol 2023HARMONY was a NEGATIVE trial - defibrotide prophylaxis did not significantly reduce SOS/VOD incidence or mortality in high-risk pediatric allo-HCT vs placebo. A...
- ABA2 JCO 2021Abatacept added to Tac/MTX significantly reduced severe (grade III-IV) acute GVHD in 7/8 MMUD allo-HCT (14.8% to 6.8%) with an acceptable safety profile, supporting...
- FORUM JCO 2021TBI plus etoposide produced significantly higher overall survival and lower relapse than chemoconditioning, establishing TBI-based conditioning as the standard for...
- REACH3 NEJM 2021Ruxolitinib significantly improved ORR and failure-free survival in steroid-refractory/dependent cGvHD compared with best available therapy, establishing it as a new...
- ROCKstar Blood 2021Belumosudil 200mg QD demonstrated a 74% ORR in heavily pretreated SR-cGVHD, including post-ibrutinib patients, with durable responses and a manageable safety profile...
- BMT CTN 1501 Blood 2020For clinical- and biomarker-defined standard-risk acute GVHD, sirolimus demonstrated similar initial treatment efficacy to prednisone while sparing steroid exposure...
- REACH1 Blood 2020Ruxolitinib produced durable responses and encouraging survival in steroid-refractory acute GVHD, supporting its FDA approval as the first agent indicated for this...
- REACH2 trial NEJM 2020Ruxolitinib significantly improved overall response and failure-free survival vs best available therapy in glucocorticoid-refractory acute GVHD, at the cost of higher...
- ATG for GVHD Prophylaxis (Kröger) N Engl J Med 2016Adding ATG to myeloablative conditioning significantly lowered chronic GVHD (32.2% vs 68.7%) without compromising relapse-free or overall survival, and improved the...
- BMT CTN 0402 Blood 2014Tacrolimus/sirolimus did not improve acute GVHD-free survival over tacrolimus/methotrexate (negative primary), but with similar long-term outcomes, faster...
- BMT CTN 1301 Blood 2014Tac/Sir did not significantly reduce acute GVHD vs Tac/MTX but was associated with significantly lower chronic GVHD and less mucositis. Increased TMA risk warrants...
- BMT CTN 0201 NEJM 2012PBSC and BM yielded equivalent 2-year OS in unrelated donor allo-HCT. PBSC offered faster engraftment and lower graft failure but significantly higher chronic GVHD....