February 12th 2025
Meta-analysis suggests iron therapy lowers the risk of heart failure hospitalization and cardiovascular death in patients with chronic kidney disease.
February 11th 2025
Iron administration was linked to a decrease in platelet counts, even in the absence of preexisting thrombocytosis.
February 7th 2025
More than 90% of patients with hemophilia B eliminated factor IX prophylaxis 4 years after treatment with etranacogene dezaparvovec (HEMGENIX) gene therapy.
Interim analysis of the Phase 3 FRONTIER3 trial found Mim8 well-tolerated in children with hemophilia A with and without inhibitors.
January 31st 2025
This FDA News Month in Review provides a round-up of regulatory decisions and submissions from January 2025.
Clinicians Challenge USPSTF Iron Deficiency Screening Recommendations in Pregnancy
Peter Kranke, MD, MBA, MME, and colleagues call for clarifications to the August 2024 recommendations with more real-world considerations.
FDA Approves Axatilimab 9 mg and 22 mg Vial Sizes for Chronic GVHD
Axatilimab in 9 mg and 22 mg vial sizes are expected to launch in early February for the treatment of chronic graft-versus-host disease.
Phase 3 Data Shows Iptacopan Eases Fatigue in C5i-Experienced, -Naïve PNH Patients
2 phase 3 trials show iptacopan improves fatigue, quality of life, and hematologic markers in C5i-experienced or -naive patients with paroxysmal nocturnal hemoglobinuria.
IV Iron Infusion Cuts Anemia Risk Before Childbirth, Study Finds
An infusion of ferric carboxymaltose in the third trimester significantly reduced anemia in pregnant women, surpassing the efficacy of iron tablets.
Iron Therapy Boosts Hemoglobin Levels in Deficient Patients with Obesity
A new study highlights notable obesity-associated disruptions of iron metabolism, with higher hemoglobin and serum ferritin levels in pre-obese and obese cohorts.
Iron Deficiency Prevalent in Acute Coronary Syndrome, Study Finds
Nearly half of patients with acute coronary syndrome in a single-center analysis exhibited iron deficiency, with anemia linked to a greater impact on mortality.
Greater Non-HDL/HDL Cholesterol Ratio Linked to Lower Anemia Rates
A study shows that a lower non-HDL/HDL cholesterol ratio is linked to greater anemia prevalence. A threshold of 4.28 shows a 15% anemia risk drop per unit increase below this value.
FDA Approves Concizumab for Hemophilia A or B with Inhibitors
Concizumab marks the first subcutaneous injection treatment for hemophilia A or B with inhibitors, demonstrating an 86% reduction in bleeds in pivotal trial data.
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.
Hematology Pipelines, Trials to Watch, with Steven Fein, MD
In this video, a hematologist discusses trials and areas of advancement he is excited about in the current landscape.
Clinically Relevant Updates in Iron Deficiency, with Steven Fein, MD
In part 2 of our 5-part series, Steven Fein, MD, discusses updates in iron deficiency management from 2024.
Recapping ASH 2024 Highlights, with Steven Fein, MD
In this video, Steven Fein, MD, discusses updates and topics of note from the American Society of Hematology annual meeting.
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.
Innovative Hemophilia Treatments Offer Hope Amid Lingering Clinical Challenges
At ASH 2024, Annette von Drygalski, MD, PharmD, described the paradigm shift that has taken place in hemophilia over the past decade.
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.
Eltrombopag Bests Standard of Care as First-Line Therapy in Pediatric Immune Thrombocytopenia
Data from the PINES trial at ASH 2024 suggest eltrombopag could outperform current standard of care for platelet response in newly diagnosed pediatric ITP.
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.
Iptacopan Boosts Hemoglobin Levels in Adults with PNH in Phase 3b Trial
Topline results from APPULSE-PNH show the positive efficacy and safety of twice-daily oral iptacopan in adults with PNH switched from anti-C5 therapies.
Dexamethasone Raises VTE Risk in COVID-19 Cases with Sickle Cell
Dexamethasone treatment was linked to worse clinical outcomes in patients with sickle cell disease hospitalized for COVID-19.