RDR Alert®

The topline phase 2b data shows both 75 mg and 150 mg doses of atacicept resulted in a 31% mean reduction in proteinuria.

The targeted patient population included those with sickle cell disease or beta thalassemia who were considered at high risk for complications, or otherwise ineligible for standard myeloablative hematopoietic stem cell transplant.

The results show similar rates o colonized C difficile among pediatric patients and adult patients with cystic fibrosis.

"The challenge right now is that we have no therapies in the emergency department to treat acute pain, outside of pain medications and IV opioids," Dr. Claudia Morris says in an interview.

The motive of the study wasn't to implement hematopoietic stem cell transplantation (HSCT) programs worldwide, but as a contribution to comprehensive cancer control efforts.

According to Dr. Corinna Schultz, a key part of the conversation of sickle cell trait (SCT) is centered around reproductive choices for the patient in the future.

The FDA's plans to expedite and expand regulatory pathways has resulted in a novel drug development boom since 2017. The commissioner discusses the details of their strategy.

Dr. Adam Lamble recommends that pediatric patients with AML and TP53 status should now be considered for transplant in first remission, and novel interventions are warranted.

In part A of the trial, 60% of patients treated with dupilumab weekly met the primary endpoint of histologic remission, compared to 5% of the placebo group.

Dr. Molly Tokaz and Dr. Lisa Force discuss the extensive data they collected on the utilization of hematopoietic stem cell transplantation (HSCT) for patients with AML.

Almost 25 years later, Dr. Claudia Morris concluded 2022 by presenting phase 2 data at the 64th ASH Annual Meeting that demonstrated the therapy's benefit, and her original premise. Her team's phase 3 trial evaluating Arg is currently underway.

Dr. Adam Lamble shares that outcomes for pediatric patients with acute myeloid leukemia that have TP53 status are inferior to other patients with high-risk disease.

Dr. Corinna Schultz talks about her latest data and the growing body of literature showing medical complications that can come with sickle cell trait.

"Using CRISPR/Cas9 you are able to actually fix a disease, that otherwise, was not fixable," Dr. Haydar Frangoul explains the data he presented at ASH 2022.

New developments in gene therapy offer a promising perspective of a future where curing sickle cell disease (SCD) is possible.

With several newly approved therapies hitting the market and an overflowing pipeline, the FDA has stepped in to help keep the burgeoning world of biotech on track.

Expert Stephanie Guarino, MD, MSHP, shares her plan for the future of pain management specifically for sickle cell disease is based on results from a series of studies.

More research presented by Dr. Stephanie Guarino resulted in a recommended guideline designed for investigating sickle cell disease pain to standardize and enhance the quality of data.

The treatment was superior in various secondary endpoints, included transfusion avoidance, changes from baseline in hemoglobin level, FACIT-F scores and ARC, and the rate of clinical BTH.

Data from the largest investigation of TP53 mutations in pediatric patients with acute myeloid leukemia (AML) is presented at the ASH 2022 Annual Meeting.

"There's no national guidance on how this information should be delivered back to families, which leaves it up to each state's newborn screening program to decide the best route that they see fit," Dr. Corinna Schultz explains.

New phase 2 interim data show rates of the erectile condition were halved after 26 weeks of treatment.

Dr. Stephanie Guarino's novel analysis found a significant relationship between self-efficacy in adults with SCD, and experiences of disease-related stigma.

Spleen size can vary significantly in pediatric patients with sickle cell anemia.

In her mitochondrial work, Dr. Claudia Morris made a novel discovery that wasn't discussed in her presentation at the ASH 2022 Annual Meeting.

The results show the mean change from baseline in PKDD was ‒7.19 for M/M patients, ‒4.58 for P/M patients, and ‒3.94 for mitapivat patients.

"Acute myeloid leukemia (AML) remains the most deadly disease among adults, with a five year overall survival of less than 30%," lead investigator Dr. Molly Tokaz says in an interview.

The results for emotional functioning, social functioning, and nausea and vomiting numerically favored D-Rd at several time points.

The accurate prediction of EF trajectory phenotypes may determine personalization of prognosis and treatment in a pediatric population with acute myeloid leukemia.

Researchers from Germany suggest the utility of platelet and absolute neutrophil counts may be misplaced in current standards for pediatric remission.