Rare Blood

The FDA has approved a gene-editing therapy for sickle cell disease (SCD) for the first time.

Iptacopan, the first oral monotherapy for patients with paroxysmal nocturnal hemoglobinuria (PNH), is now an FDA-approved option for improving hemoglobin levels.

Pregnancy-onset thrombotic thrombocytopenic purpura (TTP) is a challenging condition with 3 distinct subtypes, which should be planned for in adults with a history of TTP.

Health-related quality of life was found to be associated with poverty status through parenting stress in children with sickle cell disease (SCD).

Patients with sickle cell disease (SCD) who wore an Apple Watch and reported daily pain scores could have their pain scores accurately predicted during hospital admission for vaso-occlusive crises and after discharge.

Durable mechanistic effects and a good safety profile were found in patients who took mitapivat for sickle cell disease (SCD).

Adzynma is the first product to be approved for the treatment of the rare blood disorder that has 90% mortality when left untreated.

Acute kidney injury (AKI) was found to be common in children aged 17 years and younger who were hospitalized for sickle cell anemia.

Patients with paroxysmal nocturnal hemoglobinuria (PNH) who had suboptimal response to eculizumab can switch to pegcetacoplan for a cost-effective alternative, according to. acost analysis.

Immune thrombotic thrombocytopenic purpura (TTP) can be treated safely and effectively with caplacizumab.

A panel of experts agreed that exa-cel, a sickle cell disease gene therapy, was safe enough for clinical use, setting the stage for a potential FDA approval; the United States saw an increase in infant mortality rates for the first time in more than 2 decades; the new antiburnout campaign from the CDC asks leaders to better support health care workers.

Pharmacy workers embark on a multiday strike; Fathers are taking paid family leave to bond with their newborns; the FDA is set to evaluate a pioneering gene therapy for sickle cell disease (SCD).

Paroxysmal nocturnal hemoglobinuria (PNH) could be effectively treated by using eculizumab, which was found to be safe in patients.

Biases in care for young adults with sickle cell disease could lead to lower quality of care for these patients.

Including biomarkers for non-ADAMTS13 in routine clinical testing could benefit diagnosis and follow-up in patients with immune-mediated thrombotic thrombocytopenic purpura (TTP).

Young women with sickle cell disease have a higher prevalence of heavy menstrual bleeding and dysmenorrhea.

Patients with paroxysmal nocturnal hemoglobinuria (PNH) had decreased quality of life (QOL) and worse fatigue that could be linked to lactate dehydrogenase levels.

The 3-year relapse and mortality rates for thrombotic thrombocytopenic purpura (TTP) were 23.6% and 26.3%, predicated on platelet count.

Although children with sickle cell disease (SCD) frequently present with abdominal pain, limited gastrointestinal evaluations are performed for them.

Severe cases of COVID-19 and COVID-19–related deaths were associated with sickle cell disease.

Children with sickle cell disease who require hospitalization for vaso-occlusive crises can experience adverse effects for up to 12 months following hospitalization, a study found.

Patients with thrombotic thrombocytopenic purpura (TTP) benefited from patient-reported outcome measures to report their quality of life.

When patients with paroxysmal nocturnal hemoglobinuria (PNH) received eculizumab, survival rates increased and thromboembolic events/major adverse vascular events (TE/MAVE) decreased.

Researchers found indicators that increased the risk of thromboembolic events in patients with paroxysmal nocturnal hemoglobinuria (PNH).

Patients with polycythemia vera (PV) and essential thrombocythemia (ET) require repeated molecular testing and improved prognostic tools.

Patients who have with sickle cell disease (SCD) could have a slower processing speed and higher cerebral blood flow compared with controls.

Painful crises in sickle cell disease or sickle cell anemia could potentially be treated using imatinib as a therapeutic option, a small study found.

A simulation case for emergency medicine residents was found to be helpful as a review of the medical diagnosis and management of osteomyelitis in patients with pediatric sickle cell disease (SCD).

In a study of adolescents and young adults with sickle cell disease (SCD), researchers did not find a link between neurocognitive performance and pain and fatigue.

The pain and impact of sickle cell disease (SCD) was associated with patient social and emotional functioning in a recent study.