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In a phase 1 trial, co‑infused autologous CD19- and BCMA-targeting CAR‑T cells produced 80% LLDAS/DORIS remission at 12 weeks in 15 refractory SLE patients, with manageable toxicity and evidence of eradication of autoreactive B‑cell and pla
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 o
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
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 a
A CIBMTR registry cohort (n=10,025) found donor age predicted worse overall survival with conventional calcineurin inhibitor prophylaxis but not with posttransplant cyclophosphamide (PTCy); attenuation was driven by reduced nonrelapse morta
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 acce
Israeli scientists have developed a novel small molecule drug that targets RNA structures maintaining cancer cell immortality, providing a promising new approach to treating hard-to-cure cancers with unparalleled precision.
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 neuro
Posttransplant gilteritinib greatly benefits FLT3-ITD AML patients with NPM1 comutations, while myeloablative conditioning offers limited advantages over reduced-intensity conditioning in eradicating measurable residual disease.
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
Favism, a hereditary enzyme deficiency causing red blood cell breakdown upon exposure to certain triggers like fava beans, is preventable with proper diagnosis and management. This article explores its clinical features, diagnosis, treatmen
The phase 2 trial shows that CM313 significantly improves platelet response rates and duration with manageable safety in adults with persistent or chronic primary immune thrombocytopenia.
Prompt opioid delivery in pediatric EDs for sickle cell pain significantly lowers hospitalization rates, with the greatest benefit seen when the first dose is given within 60 minutes and the second dose within 30 minutes.
This study demonstrates the long-term effectiveness and safety of autologous gene therapy for treating ADA deficiency, showing sustained immune function and minimal adverse effects over a median follow-up of 7.5 years.
Over the past two decades, advances in therapy have doubled the median survival of multiple myeloma patients, transforming this once devastating cancer into a more manageable disease through targeted drugs and immune therapies.
Recent clinical trials report encouraging tumor shrinkage with KRAS G12D inhibitor VS-7375, significant progression-free survival benefit in soft tissue sarcoma with ozekibart, and a novel gene therapy acquisition targeting wet AMD.
This study identifies specific T-cell receptor clonotypes associated with aggressive subtypes and poorer survival in mycosis fungoides and Sézary syndrome, supporting TCR sequencing as a tool for refined risk stratification in cutaneous T-c
This randomized trial demonstrates that eltrombopag significantly improves sustained platelet response in children with newly diagnosed ITP, offering a potential early intervention option.
This study highlights the prognostic significance of tumor Ki-67 changes during neoadjuvant treatment, offering potential for improved risk stratification and personalized therapy in breast cancer patients.
A restrictive treatment initiation approach in CLL, endorsed by the Brazilian Group of CLL, identifies patients with higher risk more accurately and spares low-risk patients from unnecessary therapy, showing superior prognostic stratificati
