Allogeneic haematopoietic stem cell transplantation (alloHSCT) is safe and effective for adolescents and adults with inborn errors of immunity (IEI) with severe disease manifestations of their disease...

Donor lymphocyte infusion (DLI) prevents acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) relapses following hematopoietic stem cell transplantation. Given the life-threatening toxiciti....

A video from Talha Badar, posted on Jul 14, 2026.

A genetically altered cancer drug, based on CAR T-cell therapies, could be a big success with leukemia patients but at a staggering cost.

Key Points. Genetics and MRD allows more accurate treatment with alloHSCT or chemotherapy of adults with Ph-negative ALL.

The company is preparing to submit the results to the FDA this year

Key Points. In a randomized ph3 study, Orca-T significantly improved survival free cGVHD compared to conventional alloHSCT with Tac/MTX.In addition to lowe

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AbstractBackground. Allogeneic hematopoietic stem cell transplantation (alloHSCT) represents a major therapeutic challenge in the treatment of hematologic

The risk of Clostridioides difficile infection (CDI) is high in patients undergoing hematopoietic stem cell transplant (HSCT) due to many factors including prolonged use of antibiotics. Our institution implemented a protocol incorporating primary oral vancomycin prophylaxis (OVP) during the index HSCT admission in June 2021.MethodsThis is a retrospective review of adult patients who underwent allogeneic (allo) or autologous (auto) HSCT between 2016-2024. Patients in alloHSCT and autoHSCT cohorts were assessed in groups based on pre- compared to post-implementation of the OVP protocol (pre vs post-June 2021; no OVP vs OVP). In the OVP groups, HSCT patients received OVP 125 mg every 12 hours at initiation of bacterial prophylaxis and continued until day of discharge. The primary outcome was CDI incidence during index HSCT admission, defined as a positive CDI test plus receipt of treatment dose vancomycin or fidaxomicin. Secondary outcomes included incidence of CDI within 30- and 60-days post-HSCT, length of stay, vancomycin-resistant Enterococcus (VRE) infection, culture-confirmed infection, and gastrointestinal graft versus host disease (GI-GVHD) within 120 days.ResultsAfter screening 425 patients, we included 390 (Allo n=250 [125 OVP vs 125 no OVP], Auto n=140 [70 OVP vs 70 no OVP]). Baseline characteristics including sex, hematologic diagnosis, and HSCT-CI score were similar between groups for both cohorts. Among alloHSCT, more patients receiving OVP had peripheral blood graft source (97% vs. 88%, p=0.009) and post-transplant cyclophosphamide-based GVHD prophylaxis (97% vs. 73%, p< 0.001). Systemic antibiotic use was similar between groups in both cohorts. Median duration of OVP was 23 days (IQR 17-29) and 15 days (IQR 9-17) in the allo and autoHSCT cohorts, respectively. There was a reduced incidence of CDI with use of OVP (Allo 2.4% vs 10%, p=0.010, Auto 1% vs 4% p=0.620). There was no difference in rates of VRE or length of stay in either cohort, but among alloHSCT there was less 120-day GI-GVHD in the OVP group (4% vs 12%, p=0.02).ConclusionAmong AlloHSCT, there was a reduced incidence of CDI without an increase in VRE infection with the use of primary OVP prophylaxis. We found a similar trend among AutoHSCT patients, though larger studies are required to confirm these findings.DisclosuresAll Authors: No reported disclosures

Researchers sought to determine whether Orca-T would lead to earlier recovery following transplant and better HRQoL compared with conventional alloHSCT.

Signal Transduction and Targeted Therapy - A donor PD-1+CD8+ TSCM-like regulatory subset mobilized by G-CSF alleviates recipient acute graft-versus-host-disease

Risk stratification in myelodysplastic syndromes (MDS) is essential for clinical decision-making, yet the optimal approach to estimate risk for patients undergoing allogeneic stem cell transplantation (alloHSCT) remains uncertain. Whether dynamic changes in risk between diagnosis and post-hypomethylating agent (HMA) therapy improve prognostic accuracy beyond baseline evaluation has not been established. We retrospectively studied 176 HMA-treated patients who underwent alloHSCT, applying the Molecular International Prognostic Scoring System (IPSS-M) at both diagnosis and pre-transplant. The primary endpoint was 4-year progression-free survival (PFS). Overall, dynamic assessment did not improve prognostic performance compared with baseline evaluation. For 4-year PFS, C-indices at diagnosis versus at alloHSCT were 0.6406 versus 0.6377 (p=0.82). Patients with worsening risk after HMA experienced notably inferior outcomes, while those with apparent improvement fared no better than patients with unchanged risk (4-year PFS: 50%, 50%, and 31% for improved, unchanged, and worsening risk, respectively). Apparent IPSS-M improvement before alloHSCT yielded no gains in survival and no reduction in relapse relative to unchanged risk, a pattern consistent among TP53 wild-type patients. Moreover, clearance of TP53 mutations following HMA therapy did not translate into improved post-transplant outcomes. In summary, dynamic reassessment with IPSS-M prior to alloHSCT offers no prognostic advantage over baseline evaluation at diagnosis in HMA-treated MDS patients. Accordingly, risk reduction should not be regarded as a therapeutic goal or trial endpoint, whereas risk progression constitutes an adverse marker that may inform incorporation of post-transplant maintenance strategies or intensified conditioning regimens to improve survival.

The FDA approved treosulfan plus fludarabine for alloHSCT conditioning in acute myeloid leukemia or myelodysplastic syndrome.

Non-viral engineering can ease CAR-T cell production and reduce regulatory and cost requirements. We utilized Sleeping Beauty transposon to engineer donor-derived anti-CD19.CD28.OX40.CD3zeta T cells differentiated in cytokine-induced killer (CARCIK-CD19) for B-cell precursor acute lymphoblastic leukemia (BCP-ALL) patients relapsed after allogeneic hematopoietic stem cell transplantation (alloHSCT). We report the results of CARCIK-CD19 observed in 36 patients (4 children and 32 adults) treated according to the final recommended dose. Cytokine release syndrome of grade 2 or lower occurred in 15 patients, ICANS grade 2 in 1 patient, and late-onset peripheral neurotoxicity of grade 3 in 2 patients. GVHD never occurred after treatment with allogeneic CARCIK-CD19. Complete remission was achieved by 30 out of 36 patients (83.3%), with MRD negativity in 89% of responders. With a median follow-up of 2.2 years, the 1-year overall survival was 57.0%, and event-free survival was 32.0%. The median duration of response at 1 year was 38.6%. CAR-T cells expanded rapidly after infusion and remained detectable for over 2 years. Integration site analysis after infusion showed a high clonal diversity. These data demonstrated that SB-engineered CAR-T cells are safe and induce durable remission in heavily pretreated patients with BCP-ALL relapsed after alloHSCT. Trial registration: The phase 1/2 and phase II trials are registered at www.clinicaltrials.gov as NCT#03389035 and NCT#05252403.