Our April month in review for hematology focuses on the latest updates to the pipeline, new data on the high mortality risk in people with sickle cell disease (SCD), and our multimedia offerings across the space.

In particular, this review focuses on the latest updates to the hematologic pipeline, mortality risk faced by people with sickle cell disease, and our newest multimedia offerings in the field, including an interview with an SCD expert and a quiz to test your knowledge on the gastrointestinal evaluation of iron deficiency anemia.

Pipeline Updates

Bitopertin Meets Primary Phase 2 Endpoint in Erythropoietic Protoporphyria

Topline data from the phase 2 AURORA study revealed the benefit of bitopertin in patients with erythropoietic protoporphyria (EPP), a rare disease caused by mutations that affect heme biosynthesis and results in the accumulation of protoporphyrin IX.

In AURORA, bitopertin led to dose-dependent, statistically significant reductions in PPIX, compared with placebo, across the 20 and 60-mg dosing cohorts. The treatment improved measures of sunlight tolerance in both cohorts, but it did not reach statistical significance, owing to a strong placebo performance.

Rilzabrutinib Achieves Phase 3 Primary Endpoint in Immune Thrombocytopenia

Meanwhile, rilzabrutinib achieved the primary endpoint of durable platelet response in adult patients with persistent or chronic immune thrombocytopenia (ITP) in the pivotal phase 3 LUNA 3 study. The oral, reversible, covalent BTK inhibitor, taken twice daily at 400 mg, led to a significantly higher proportion of patients with ITP who achieved a durable platelet response, versus a placebo-treated cohort.

“The results of this study reinforce rilzabrutinib’s potential to be a first-in-class oral, reversible BTK inhibitor that can provide clinically meaningful improvements for people living with severe immune-mediate diseases like ITP,” Houman Ashrafian, executive vice president and head of research and development at Sanofi, said in a statement.

Sickle Cell Disease

Patients with SCD Face Early Death Despite Improved Survival Rates

A retrospective, multicenter study assessed risk factors linked to premature mortality among children and adults with sickle cell disease (SCD) admitted between 2006 and 2020. Factors, including male gender, low fetal hemoglobin, rapid drop in hemoglobin and platelet counts, and increases in white blood cell count, LDH, ferritin, and C-reactive protein, correlated significantly with mortality in patients with SCD.

“In summary, the ability to identify the risk factors that are associated with increased mortality among SCD patients permits accurate prognostication and provides effective prophylactic management strategies,” investigators wrote.

Frequent Hospitalized VOCs Worsens Mortality Risk in Sickle Cell Disease

A large nationwide study in France found the likelihood of mortality in SCD increased with the number of hospitalized vaso-occlusive crises (HVOCs) experienced one year before death.

Individuals with SCD endure frequent VOCs and early mortality—this analysis showed a notable association between HVOC and death and multiple hospitalized complications, with the strongest associations for acute chest syndrome, sepsis, acute kidney injury, osteonecrosis, and pulmonary embolism.

“Better management of HVOCs is needed not only to improve the patient’s quality of life, but also to prevent premature mortality, and debilitating complications,” investigators wrote.

Multimedia Offerings

Elna Saah, MD: Unraveling the Current Landscape in Sickle Cell Disease

In a recent interview with HCPLive Hematology, Elna Saah, MD, a hematologist at the Emory University School of Medicine, described the current care landscape in SCD, the burdens as more children with the condition grow older, and balancing pain management with the opioid crisis.

“It’s a real big problem, a public health issue because it is progressive and complications continue to evolve as patients get older and older,” Saah said. “In adults, there’s a lot of multiple organ involvement, which reduces the lifespan, causes earlier mortality and morbidity, and burdens both the patient and the healthcare system.”

Clinical Quiz: Gastrointestinal Evaluation of Iron Deficiency Anemia

The American Gastrointestiological Association (AGA) released updated guidelines in 2020 for the gastrointestinal evaluation of iron deficiency anemia. The 10-page document provides specific recommendations for anemia, including the cutoff of ferritin levels in diagnosis, bidirectional endoscopy, and differences in anemia diagnosis by sex.

In our latest clinical quiz on the document, we test your knowledge of the recommendations for gastrointestinal evaluation of IDA based on the AGA 2020 guidelines.