RDR Alert®

This past year has brought unique challenges for sickle cell disease patients and communities of color. The ongoing vaccination campaign is yet another challenge that healthcare communities must find ways to overcome.

A sickle cell expert reflects on the state of care and access for sickle cell communities.

Younger individuals and those with specific sickle cell genotypes led to earlier predictions for fetal risk of sickle cell disease.

Older age, macroalbuminaria, and high diastolic blood pressure are among some of the risk factors significantly associated with onset of chronic kidney disease.

More studies are needed to determine whether bi-level positive airway pressure ventilation can help prevent acute chest syndrome.

Data from the large-scale, population-based study corroborate previous single-center study findings.

Elexacaftor/tezacaftor/ivacaftor and ivacaftor was previously approved for CF patients at least 12 years old.

COVID-19 severity should be looked at in the context of pre-existing comorbidities, age, social determinants, and bias among data sources.

The new indication for the therapy provides coverage for patients with the rare blood disease as young as 1 month old.

Treatment with plasminogen, human-tmvh was associated with improvements in existing lesions and prevention of new lesions in supporting data.

There are no guidelines on sickle cell patient referral to pulmonologists, associated outcomes, and best therapeutic interventions.

Jordana Hoppe, MD, highlights some key points about the genetic condition.

Resolution of iron deficiency was associated with lower mortality; however, mortality risk was similar following resolution of anemia.

All patients with severe disease had abnormal ECG readings.

Jordana Hoppe, MD, discusses the burden of cystic fibrosis on infants and children.

There was a notable association between the comorbid population and congestive heart failure exacerbations.

Social determinants can play a large role in hindering patient readiness for adult care.

CX-01 added to standard of care was well-tolerated and did not increase incidence of bleeding in patients >59 years old.

There was an association between exercise time and increased ejection fraction.

Pegcetacoplan outperformed competitor in a phase 3 head-to-head study.

The single 1000 mg dose treats iron deficiency anemia in adult patients.

Sickle cell patients in rural areas may actually benefit from telemedicine practices and access to satellite clinics.

While obesity is less common in pediatric sickle cell patients, BMI may impact airway resistance in this population.

Early results from the MOMENTUM study show that treated patients experienced fewer vaso-occlusive episodes and related hospital admissions.

A podcast interview with study author Paul Yang, MD, PhD, on the current research and future implementation of the agent.

The AAV8 vector–based gene therapy was associated with improvements in 2 measures of visual function.

The twice-daily oral agent is indicated for patients ≥3 years of age

Not all individuals with sarcoidosis need to be treated.

Data also suggests the nonionic block polymer surfactant may be harmful for children <16 years of age.

Ennis James, MD, discusses the challenges and lingering questions associated with sarcoidosis