Victoria Johnson joined the MJH Life Sciences in 2020 and has written for NeurologyLive, CGTLive, and now HCPLive. You can reach her at vjohnson@mjhlifesciences.com
FDA Approves First Acellular Tissue Vessel Therapy for Extremity Vascular Trauma
The ATEV therapy, named Symvess, provides revascularization when autologous vein graft or implantation of a synthetic graft is not feasible.
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Colchicine Prophylaxis for Gout Flares Linked to Lower Cardiovascular Events
These findings, from an observational study, should be confirmed in a randomized, controlled trial.
Julia Weisinger, MD: Obinutuzumab a Promising Option for Intolerant/Refractory iTTP
Weisinger discussed findings from the largest cohort of patients with iTTP treated with Obinutuzumab.
FDA Approves Seventh Ustekinumab Biosimilar
Celltrion’s CT-P43/ustekinumab-stba has been approved under the name Steqeyma and is expected to launch in February 2025.
Tadashi Matsushita, MD, PhD: Reducing Treatment Burden in Hemophilia A
Matsushita discussed findings from the OLE FRONTIER4 study of Mim8.
Davide Matino, MD, MSc: Bringing Marstacimab Treatment to Hemophilia A and B
Matino discussed data from the phase 3 BASIS study evaluating the recently approved Hympavzi.
Ben Samelson-Jones, MD, PhD: Validating Long-Term Safety of Hemophilia AAV Gene Therapy
Samelson-Jones discussed follow-up data of up to 6 years with investigations of fidanacogene elaparvovec.
Françoise Bernaudin, MD: A Decade of Follow-up Reveals allo-SCT Superiority Over SOC for Sickle Cell Anemia
Bernaudin discussed the Drepagreffe-1 and 2 studies and improvements seen over 10 years of follow-up.
Pegcetacoplan Demonstrates Control in Both C5i-Experienced and Naïve PNH
Real-world clinical data from the Adelphi PNH II Disease Specific Programme were reported at ASH 2024.
Danicopan a Safe Option for Breakthrough Bleeds in PNH
Danicopan add-on therapy demonstrated more favorable safety in the ALPHA trial than pegcetacoplan did in the PEGASUS trial in a new analysis.
Achieving Quick Responses in Sickle Cell Anemia With Early, Appropriate Hydroxyurea Dosing, with Abena Appiah-Kubi, MD, MPH
Appiah-Kubi also noted that despite elevated HbF levels, very young children with SCA already had anemia and reticulocytosis.
Steven W. Pipe, MD: Fitusiran With Anti-Thrombin Modulation Yields Effective Bleed Control, Reduces Infusions for Hemophilia
Pipe discussed findings from the open-label extension of the ATLAS studies at ASH 2024.
Highlighting the Danger of SCI Progression during iTTP Remission, with Shruti Chaturvedi, MBSS, MS
Chaturvedi discussed findings from the NeST study at ASH 2024, including an increased risk of stroke in those with silent cerebral infarction progression.
Caroline Piatek, MD: Improving Patient-Reported Outcomes in PNH With Danicopan Add-on Therapy
Piatek shared the latest data update from the phase 3 ALPHA study at the ASH 2024.
Haydar Frangoul, MD: Preventing VOCs in People With Sickle Cell Disease With Exa-Cel Gene Editing Therapy
Frangoul discussed highlights from the latest data update from the CLIMB SCD-121 trial of Casgevy.
Hydroxyurea Shows Clinical Benefit in HbSC, Misses the Mark on Safety
While the therapy was generally well-tolerated, the PIVOT trial failed its primary endpoint of dose-limiting toxicities.
Etavopivat Well-Tolerated With Some Disease-Modifying Activity in SCD
The therapy uses a novel pyruvate kinase activator mechanism and warrants further investigation in an ongoing phase 3 trial.
Rilzabrutinib Improves Platelet Response, Quality of Life in Immune Thrombocytopenia Compared to Placebo
Notably, participants without platelet responses also experienced improvements in fatigue, which may be due to the monoclonal antibody’s anti-inflammatory action.
Rheumatology Month in Review: November 2024
The rheumatology month in review emphasizes new data from ACR 2024 in lupus, gout, and fibromyalgia.
Biosimilars Month in Review: November 2024
The biosimilars month in review highlights new equivalency data for denosumab and tocilizumab biosimilars.
Tocilizumab Biosimilar Demonstrates Similar Safety, Efficacy for RA to Reference
Pharmacokinetics and immunogenicity were also similar between reference tocilizumab and CT-P47.
Cell Therapy Gets RMAT Designation for HLHS, Improves Survival in Heart Failure With Reduced Ejection Fraction
Phase 1/2 data prompted the RMAT designation for HLHS shortly after Mesoblast announced positive phase 3 data for HFrEF.
Women Who Give Birth After Infertility Have Elevated Risk of Systemic Rheumatic Disease
Women who gave birth after infertility without receiving fertility treatments had an elevated risk of developing SARDs, such as lupus.
Orrin Troum, MD: Accurately Imaging Gout With DECT Scanning
Troum discussed the AGILE study and how DECT scanning helped visualize improvements in uncontrolled gout.
Patient Education Materials for Rheumatology, Internal Medicine Don’t Meet Readability Standards
CDC, NIH, and AMA recommend PEMs to be at a 6th-to-8th-grade reading level, while assessed materials were readable at high school or higher levels.
Denosumab Biosimilar Demonstrates Equivalent Efficacy, Safety in Phase 3 Trial
CT-P41 had similar rates of bone mineral density increases and adverse events in data presented at the ACR 2024 Convergence.
CAR-T Therapy Gets Fast Tracked for Relapsed/Refractory SLE
The GLEAM trial is currently enrolling patients to receive SC291, with data expected in 2025.
FDA Approves Ustekinumab (STELARA) Biosimilar for Crohn’s, UC, Psoriasis, PsA
Ustekinumab-kfce is planned for launch in February 2025 according to a previous settlement and license agreement with Janssen.
Real-World Study Confirms Similar Efficacy of Guselkumab and IL-17i for PsA
Findings from PsABIOnd can help reassure clinicians of similar efficacy between treatment choices.
PEGylated Uricase Well-Tolerated, Demonstrates Dose-Dependent Urate-Lowering
At the highest dose levels, mean plasma urate levels remained below 6.0 mg/dL for up to 12 weeks.