Rare Blood

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).

Kevin Davies, PhD, executive editor, The CRISPR Journal and GEN Biotechnology, and author of Editing Humanity: The CRISPR Revolution and the New Era of Genome Editing, discusses the difficulties inherent in making CRISPR gene editing technology an equitable treatment.

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.

Patients with acquired thrombotic thrombocytopenic purpura (aTTP) showed significant benefits in critical outcomes when treated with caplacizumab as well as the standard regimen.

By giving their patient the correct diagnosis, clinicians avoided costlier treatments for thrombotic thrombocytopenic purpura (TTP) like plasmapheresis.

Congenital thrombotic thrombocytopenic purpura (TTP), an ultra-rare disease, can significantly impact quality of life, but clinicians have previously lacked a disease-specific way to track experiences via patient-reported outcomes (PROs).

The researchers noted that their paper is the first, to their knowledge, to report on a case of thrombocytopenic purpura (TTP) in a patient with metastatic renal cell carcinoma (RCC).

Study authors say the consequences of misdiagnosing osteomyelitis can be severe in the disease setting of sickle cell disease (SCD).

A new review article illuminates why physicians should keep coagulation disorders in mind when evaluating patients with heavy abnormal uterine bleeding.

Black children with sickle cell disease (SCD) face a disproportionate burden of social determinants of health (SDOH), the study found.

Investigators stress the importance of ongoing research on the association between depression and pain in patients with sickle cell disease (SCD).

Artificial intelligence may be leveraged to improve minimal residual disease measurements for patients.

Approximately 2500 individuals in the United States could be eligible to receive valoctocogene roxaparvovec.

Authors suggest the finding may indicate the cells play a role in hematological malignancies.

The infrastructure also provides a blueprint for similar initiatives in other rare diseases.

A recent review highlighted a selection of emerging therapies and advances in sickle cell disease (SCD) that focus on renal manifestations.

The study also highlighted disparities in accurate diagnosis of myelodysplastic syndromes (MDS).

Skin rashes are common among patients who complete hematopoietic cell transplantation (HCT).