Jul 14th, 2021 - Alpha1-antitrypsin deficiency (AATD) is caused by mutations in the SERPINA1 gene, coding for alpha1-antitrypsin (AAT). AAT is synthesised mainly in the liver and is released into bloodstream to protect tissues (particularly lung) with its antiprotease activity. The homozygous Pi∗Z mutation (Pi∗ZZ genotype) is the predominant cause of severe AATD. It interferes with AAT secretion thereby leading...

Aug 3rd, 2020 - This new edition of the World Federation of Hemophilia (WFH) guidelines for the management of hemophilia comes at an exciting time in the evolution of the diagnosis and treatment of this condition. Since the publication of the second edition in 2012, tremendous advances have been made in several aspects of the management of hemophilia. These include genetic assessment as well as therapy with ma...

Jul 8th, 2020 - In a small sample, retreatment of patients with large B-cell lymphoma showed good response, notably in patients who had complete response on initial treatment, according to a poster presented at ASCO 2020 Virtual Scientific Program.

Jul 6th, 2020 - A novel CAR T-cell therapy targeting both CD19 and CD22 showed less toxicities than current CAR T-cell therapies while producing complete response with accepted regimens, according to a study presented at ASCO2020 Virtual Scientific Program.

Jul 2nd, 2020 - CAR T-cell therapy with BCMA-targeting idecabtagene-vicleucel (ide-cel) has emerged to be a game-changer as shown by this first pivotal phase II trial of CAR T-cell therapy in multiple myeloma (MM). Ide-cel is a first-in-class BCMA-directed CAR T-cell therapy, with 4-1BB costimulatory domain and CD3Z T-cell activation domain.

Jun 27th, 2020 - The coronavirus disease 2019 (COVID-19) pandemic caused by SARS coronavirus 2 (SARS-CoV-2) has caused significant morbidity and mortality for patients and stressed healthcare systems worldwide. The clinical features and outcomes of COVID-19 among immunosuppressed patients, who are at presumed risk for more severe disease but who may also have decreased detrimental inflammatory responses, are no...

Jun 24th, 2020 - Awaited results of the ENDURANCE trial showed equivalency between a regimen of carfilzomib plus lenalidomide and dexamethasone (KRd) and bortezomib, lenalidomide, and dexamethasone (VRd) as an initial therapy for patients with standard- or intermediate-risk multiple myeloma (Abstract LBA3). KRd failed to improve progression-free survival compared with VRd, according to data presented by Shaji K...

Jun 24th, 2020 - For patients with stage IV metastatic breast cancer and an intact primary tumor (ITP), the early initiation of locoregional treatment (LRT) was not associated with longer survival or improvements in health-related quality of life (HRQoL) compared with systemic therapy alone, according to a phase III clinical trial (Abstract LBA2). These findings appear to add to a robust evidence base that sugg...

Jun 24th, 2020 - A switch maintenance therapy approach using the immune checkpoint inhibitor avelumab in patients with advanced urothelial carcinoma (UC) who had not progressed after induction platinum-based chemotherapy offered significantly improved survival outcomes, according to results of the JAVELIN Bladder 100 trial (Abstract LBA1). The study, which was the first UC study ever presented during an ASCO An...

Jun 1st, 2020 - Alberto F. Sobrero, MD, of the Ospedale San Martino, discusses final results of the IDEA study, which supported the use of 3 months of adjuvant CAPOX, vs 6 months, for most patients with stage III colon cancer. The shorter treatment duration reduced toxicity, inconvenience, and cost (Abstract 4004).

Jun 1st, 2020 - Rana R. McKay, MD, of the University of California, San Diego, discusses the results of a phase II trial of intense neoadjuvant hormone therapy followed by radical prostatectomy in men with high-risk prostate cancer. The data show that 21% of patients had a favorable pathologic response (Abstract 5503).

Jun 1st, 2020 - Early local therapy did not prolong survival among women with de novo metastatic breast cancer and intact primary tumors, according to randomized phase 3 study results presented during the ASCO20 Virtual Scientific Program.

Jun 1st, 2020 - An allogeneic chimeric antigen T-cell therapy plus an anti-CD52 monoclonal antibody appeared safe for certain patients with large B-cell or follicular lymphoma, according to study data presented during the ASCO20 Virtual Scientific Program.

Jun 1st, 2020 - Combination therapy with the isocitrate dehydrogenase 1 (IDH1) inhibitor ivosenidib plus the BCL2 inhibitor venetoclax with or without the chemotherapeutic agent azacitidine showed activity in patients with IDH1-mutated acute myeloid leukemia (AML) in a phase Ib/II trial. The results of the study—presented by Lachowiez et al during the Hematologic Malignancies Oral Abstract Session of the ASCO2...

Jun 1st, 2020 - Women who present at diagnosis with advanced breast cancer have faced an unanswered question: will local therapy, consisting of surgery and radiation to the tumor in the breast, prolong survival compared to the traditional treatment of systemic treatment alone?

Jun 1st, 2020 - Eric Jonasch, MD, of The University of Texas MD Anderson Cancer Center, discusses phase II study findings on the oral HIF-2α inhibitor known as MK-6482, which showed efficacy and tolerability in patients with Von Hippel-Lindau (VHL)–associated clear cell renal cell carcinoma as well as responses in other VHL-related lesions (Abstract 5003).

Jun 1st, 2020 - Seema A. Khan, MD, MPH, of the Lynn Sage Comprehensive Breast Center, discusses phase III trial results showing that in newly diagnosed metastatic stage IV breast cancer, locoregional treatment of the primary tumor did not offer a greater survival benefit than systemic therapy (Abstract LBA2).

May 31st, 2020 - Neither fulvestrant alone nor fulvestrant plus anastrozole improved the endocrine-sensitive disease rate (ESDR) versus anastrozole alone as neoadjuvant endocrine therapy in postmenopausal women with locally advanced estrogen receptor-positive HER2-negative (ER+ HER2–) breast cancer in the phase 3 ALTERNATE trial.

May 31st, 2020 - Doxorubicin in combination with trabectedin appeared to be an effective and safe first-line therapy for metastatic leiomyosarcoma, according to results of the phase 2 LMS-02 study presented during the ASCO20 Virtual Scientific Program.

May 31st, 2020 - Almost 100% of patients with relapsed/refractory indolent non-Hodgkin lymphoma (iNHL) responded to treatment with the chimeric antigen receptor (CAR) T-cell therapy axicabtagene ciloleucel (axi-cel, Yescarta), data from an ongoing study showed.