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.
Mitapivat Achieves Phase 3 Endpoint in Non-Transfusion-Dependent Thalassemia
In the ENERGIZE trial, oral mitapivat achieved meaningful improvements in symptoms of anemia in patients with non-transfusion-dependent thalassemia.
Exa-Cel Shows Long-Term Clinical Benefit in Sickle Cell Disease, TDT
The CRISPR-based gene therapy demonstrated durable effects on SCD and TDT, with the longest follow-up extending past 5 years.
Reni-Cel Displays Clinical Benefit for Transfusion-dependent Beta Thalassemia
Phase 1/2 EdiTHAL trial data shows reni-cell remained well-tolerated and displayed promising efficacy for patients with TDT.
RUBY: Reni-Cel Displays Promising Efficacy, Safety for Sickle Cell Disease
Phase 1/2/3 trial data show the investigational gene-editing medicine has been well-tolerated, with encouraging efficacy, for people living with SCD.
Menstrual Blood Loss Determines Iron Status in Premenopausal Blood Donors
Blood loss due to whole blood donation and menstruation were the two most important determinants for ferritin and hemoglobin levels in female blood donors.
Adults with Sickle Cell Report Poor Health-Related Quality of Life Scores
Based on the 36-item Short Form Survey questionnaire, adults with SCD in France report low physical and mental health summary scores.
Hydroxyurea Therapy May Affect Fertility in Sickle Cell Disease
Hydroxyurea treatment may impact seminal fluid levels and ovarian reserves in male and female patients with sickle cell disease.
Iron Deficiency Prevalence Differs According to Definition Used for Women
A higher serum ferritin threshold could lead to better diagnosis and treatment of more women with iron deficiency.
Knowledge Levels, Adherence to Medical Advice Critical in Anemia Care
Knowledge, attitudes, and practices remain relevant for disease management among patients with anemia.
High-Impact Chronic Pain in Sickle Cell Linked to Poor Health Outcomes
High-impact chronic pain is a severely affected subgroup in SCD and is associated with greater pain burden and worse health outcomes.
Significant Fatigue Common in Adult Patients with Sickle Cell Disease
A lower FACIT-Fatigue score, indicating higher fatigue was independently associated with female sex, a high BMI, a high stress level, and poor sleep quality.
Daprodustat Exhibits Dose-Dependent Correlation with Hemoglobin in Anemia
Daprodustat 25-30 mg was linked to the most significant rise in serum hemoglobin and reduced serum ferritin levels.
MITIGATE: Inebilizumab Reduces Risk of IgG4-Related Disease Flare
Topline results from the randomized, placebo-controlled Phase 3 clinical trial demonstrated a statistically significant 87% reduction in IgG4-RD flares.
Hematology Month in Review: May 2024
Our May 2024 month-in-review for hematology focuses on important FDA updates, new anemia data from ERA 2024, and the latest imaging modalities.
Quality Improvement Project Increased Stroke Screening in Children with SCD
A quality improvement initiative exhibited a sustained increase in transcranial Doppler screening rates over an 8-year intervention period.
Anemia Diagnostic Tests Infrequent, Linked to Low Treatment Rates in CKD
Diagnostic tests for anemia etiology are infrequently ordered, leading to a significant majority of patients with CKD receiving inadequate treatment.
Mortality Risk Unchanged by Iron Dose in Hemodialysis Patients with Cancer
Iron administration did not increase the mortality risk of hemodialysis patients with cancer, but the safety of increasing ESA doses was not confirmed.
Anemia Increases Risk of End-Stage Kidney Disease in IgA Nephropathy
An analysis of more than 800 IgAN patients treated from 2002-2021 provides an overview of factors associated with anemia as well as its impact on prognosis.
SGLT2 Inhibitors Can Improve Anemia in Patients with CKD
Data from ERA 24 builds on previous research examining the effect of SGLT2 inhibitors on hemoglobin levels among patients with chronic kidney disease.
Smartphone App Accurate in Anemia Detection from Conjunctival Images
The smartphone app demonstrated higher accuracy, sensitivity, and specificity in patients with severe anemia than those with moderate anemia.
Intravenous Immunoglobulin May Benefit Severe Autoimmune Hemolytic Anemia
Retrospective data suggest the off-label use of IVIg could be helpful in the management of severe autoimmune hemolytic anemia.
Oral Liposomal Iron Helps Correct Iron Deficiency in Non-Dialysis CKD
Liposomal iron achieves a partial correction of transferrin saturation, with no significant effect on iron storage and hemoglobin, in patients with NDD-CKD.
Artificial Intelligence Algorithms Detect Anemia from Fundus Images
Automated anemia detection from fundus images may benefit patients undergoing routine retinal imaging.
FDA Clears IND Application for CID-103 in Immune Thrombocytopenia
Announced on May 15, 2024, the IND application supports a phase 1/2 study of CID among adults with chronic ITP.
Age, Genetics Help Predict Proliferative Sickle Cell Retinopathy
Age and sickle cell disease genotype predicted individuals with sickle cell retinopathy at risk for proliferative disease.
Anti-VEGF Improves Outcomes in Proliferative Sickle Cell Retinopathy
Within one month of treatment, anti-VEGF therapy improves anatomic and visual outcomes among eyes with stage 3 and 4 proliferative sickle cell retinopathy.
FDA Updates ALTUVIIIO Label with Full Pediatric Data in Hemophilia A
Announced by Sanofi on May 10, 2024, the updated FDA label is based on full results from the pivotal phase 3 XTEND-Kids study.
Hydroxyurea Dose Escalation Effective for Children with Sickle Cell Anemia
Extended follow-up of a phase 1/2 trial finds hydroxyurea dose optimization significantly improves clinical responses in children in sub-Saharan Africa with sickle cell.
Hematology Month in Review: April 2024
Our April month in review for hematology breaks down the latest updates to the pipeline, mortality risk in people with sickle cell disease, and our newest multimedia offerings.
Recombinant ADAMTS13 Prophylaxis Effective Approach for Congenital TTP
An interim analysis of a phase 3 trial displays the effectiveness of prophylaxis with recombinant ADAMTS13, achieving approximately 100% of normal ADAMTS13 levels.