Rare Blood

The multinational, open-label, controlled, randomized, crossover, phase 3 TAK-755 (NCT03393975) trial demonstrated superior efficacy and safety of recombinant ADAMTS13 as prophylaxis in patients with congenital thrombotic thrombocytopenic purpura (cTTP) compared with standard therapy.

An analysis of 2 phase 3 trials supporting the use of pegcetacoplan (PEG) in the treatment of paroxysmal nocturnal hemoglobinuria (PNH) assessed the benefits of this treatment compared with eculizumab (ECU) and supportive care measures.

A cross-sectional analysis assessed the birth prevalence of sickle cell disease (SCD) alongside data on social vulnerability to highlight the needs of communities impacted by SCD.

A retrospective analysis suggested that immune cells play a role in the progression of paroxysmal nocturnal hemoglobinuria (PNH).

A team of researchers developed 3 quality improvement measures embedded in electronic health records (EHRs) to improve care delivery in patients with heparin-induced thrombocytopenia.

An ongoing, phase 3, open-label study demonstrated the efficacy of exagamglogene autotemcel (exa-cel) infusion to promote transfusion dependence in patients with transfusion-dependent β-thalassemia (TDT).

A cost-utility analysis conducted from the perspective of the Italian health system found that pegcetacoplan was more effective and less costly than 2 complement 5 (C5) inhibitors for the treatment of paroxysmal nocturnal hemoglobinuria (PNH).

In a registry-based analysis, more hospitalized children with SCD and COVID-19 had severe infection requiring supplemental oxygen, compared with the general population.

A retrospective cohort study assessed patterns of opioid use in children with sickle cell disease (SCD) following their initial opioid prescription for pain management, finding that concerning patterns of opioid abuse did not develop.

A retrospective study reveals that measures of cognitive function, specifically processing speeds, decline in individuals with sickle cell disease as they grow older.

The first-in-class oral factor D inhibitor danicopan (Voydeya) has been approved for the management of extravascular hemolysis in patients with paroxysmal nocturnal hemoglobinuria (PNH).

A retrospective, qualitative cohort study concludes that patients with sickle cell disease (SCD) may have been negatively impacted by the 2016 CDC guidance.

A descriptive, qualitative study investigated the experiences and perspectives of patients with venous thromboembolism on shared decision-making and treatment with anticoagulants.

A single-center, retrospective study examined the management of thrombotic events in patients presenting with thrombocytopenia following vaccination against COVID-19.

A single-center, retrospective study demonstrated the major success of immunosuppressive therapies in contributing to complete remission in patients with acquired hemophilia A.

A cross-sectional study analyzing morphological and dimensional features of palatal rugae patters revealed that distinct anomalies exist in children with sickle cell disease (SCD) compared with children without.

A meta-analysis on complement inhibitors in the management of paroxysmal nocturnal hemoglobinuria (PNH) reveals an association between these interventions and positive treatment outcomes.

Pain screening in young patients with sickle cell disease (SCD) has demonstrated benefits for the administration of integrative health and medicine modalities for pain management.

A cross-sectional comparative study demonstrated the increased prevalence of hearing impairment in children and adults with sickle cell disease (SCD) or sickle cell traits compared with healthy individuals.

A state-wide analysis draws attention to the fact individuals with sickle cell disease (SCD) completed a vaccination against COVID-19 at nearly half the rate of individuals without SCD.

A retrospective analysis surveys the treatment landscape and patterns of patients with paroxysmal nocturnal hemoglobinuria (PNH), illuminating high hospitalization rates and emergency department use despite the availability of multiple treatment options.

A myeloid panel mutation analysis should be prioritized for patients with clonal cytopenia of undetermined significance (CCUS) as it can better assess patient risks for progression to a myeloid malignancy, a recent study argues.

Patients with hemophilia A experienced prognostic and quality of life benefits from emicizumab (Hemlibra; Genentech) prophylaxis treatment.

A single-center study suggests complement inhibitors do not reduce the severity of a COVID-19 Omicron infection in patients with paroxysmal nocturnal hemoglobinuria (PNH).

“These autosomal diseases, from an inheritance standpoint, affect men and women equally, but because of their impact on the gynecological system and menses, women are often affected much more than men," hematologist Donna DiMichele, MD, explains.

The top 5 articles about rare blood featured information on pseudo–thrombotic thrombocytopenic purpura (TTP), immune TTP, and hemophilia.

Patients with immune-mediated thrombotic thrombocytopenic purpura (TTP) were found to have white matter damage in the frontal lobe and cingulate cortex that could affect short-term memory and depressive scores.

Patients who had concurrent thrombotic thrombocytopenic purpura (TTP) and COVID-19 had elevated risk of mortality and other complications.

Children with sickle cell disease and their caregivers experience an increased burden on health-related quality of life (HRQoL) due to the frequency of vaso-occlusive crises (VOCs).

A medication monitoring program using medication bottles waws found to be effective in patients with sickle cell disease (SCD), though the usage will need additional support.