Allogeneic HCT Rebounds, PTCy Reshapes Donor Choice, and CAR‑T Use Surges: Key Trends from the 2013–2023 CIBMTR Activity Report

Allogeneic HCT Rebounds, PTCy Reshapes Donor Choice, and CAR‑T Use Surges: Key Trends from the 2013–2023 CIBMTR Activity Report

CIBMTR's 2013–2023 registry update shows a 2023 rebound in allogeneic HCT—driven by older adults—widespread adoption of post‑transplant cyclophosphamide (PTCy) across donor types, increasing mismatched donor use, and rapid expansion of commercial CAR‑T therapy; relapse remains the leading cause of death.
Donor CMV Seropositivity Tied to Worse Survival in CMV‑Seronegative AML Patients Undergoing Unrelated HCT with Post‑Transplant Cyclophosphamide

Donor CMV Seropositivity Tied to Worse Survival in CMV‑Seronegative AML Patients Undergoing Unrelated HCT with Post‑Transplant Cyclophosphamide

In CMV‑seronegative adult AML recipients receiving unrelated donor hematopoietic cell transplantation with PTCy, donor CMV seropositivity was associated with worse overall survival—primarily through a trend toward higher relapse—supporting consideration of donor CMV serostatus in selection.
Isolated HLA‑DQB1 Mismatch and Donor Age Do Not Worsen Survival After Unrelated Donor HCT with Post‑Transplant Cyclophosphamide: A Single‑Center Analysis

Isolated HLA‑DQB1 Mismatch and Donor Age Do Not Worsen Survival After Unrelated Donor HCT with Post‑Transplant Cyclophosphamide: A Single‑Center Analysis

A single‑center retrospective study of 988 unrelated donor HCTs with PTCy found no significant differences in survival or relapse for isolated HLA‑DQB1 mismatch, 7/8 mismatches, or older donor age; 7/8 mismatch increased severe acute GVHD and donor age modestly raised risk of grade II–IV aGVHD.
Non‑conditioned Autologous Gene Therapy Reverses Bone Marrow Failure in Fanconi Anaemia‑A: FANCOLEN‑1 Phase 1/2 and Long‑Term Outcomes

Non‑conditioned Autologous Gene Therapy Reverses Bone Marrow Failure in Fanconi Anaemia‑A: FANCOLEN‑1 Phase 1/2 and Long‑Term Outcomes

The FANCOLEN‑1 trial shows that infusion of autologous FANCA‑corrected CD34+ cells without cytotoxic conditioning produced sustained engraftment and clinical improvement in a majority of treated patients with Fanconi anaemia‑A, with an acceptable short‑term safety profile and no genotoxicity detected during follow‑up.
Restrictive Red Blood Cell Transfusion Is Safe for Most Patients — Except in Neurocritical Care (and Some Bleeding Syndromes)

Restrictive Red Blood Cell Transfusion Is Safe for Most Patients — Except in Neurocritical Care (and Some Bleeding Syndromes)

A Cochrane update (2025) of 69 randomized trials found restrictive RBC transfusion thresholds (typically Hb 7–8 g/dL) cut transfusion exposure ~42% without increasing 30‑day mortality overall, but liberal strategies improved long‑term neurological outcomes after brain injury and restrictive thresholds reduced mortality in GI bleeding.
Belantamab Mafodotin Plus Bortezomib and Dexamethasone Significantly Improves Overall Survival in Relapsed/Refractory Multiple Myeloma: Updated DREAMM-7 Results

Belantamab Mafodotin Plus Bortezomib and Dexamethasone Significantly Improves Overall Survival in Relapsed/Refractory Multiple Myeloma: Updated DREAMM-7 Results

The DREAMM-7 phase 3 trial shows that belantamab mafodotin combined with bortezomib and dexamethasone produces early, sustained, and statistically significant overall survival and deep, durable responses versus daratumumab-based therapy in relapsed/refractory multiple myeloma.
Selective JAK2 Inhibition OB756 Shows Promise in Hydroxyurea/Interferon‑Intolerant or -Resistant Essential Thrombocythemia

Selective JAK2 Inhibition OB756 Shows Promise in Hydroxyurea/Interferon‑Intolerant or -Resistant Essential Thrombocythemia

A Phase II multicenter study reports that OB756, a selective JAK2 inhibitor, is active and reasonably well tolerated in hydroxyurea- or interferon-intolerant/resistant essential thrombocythemia, producing platelet and leukocyte control, spleen volume reduction, symptom improvement and reductions in JAK2 V617F allele burden.