Rose McNulty

Sufficiently powered studies are necessary to investigate associations between clinical measures and patient-reported outcomes (PROs) in children with sickle cell disease experiencing acute pain episodes, according to a recent study.

A panel of specialists proposed 26 essential elements for comprehensive pediatric sickle cell disease care, laying groundwork for standardized guidelines and the establishment of accredited care centers in the future.

While chronic lymphocytic leukemia (CLL) typically progresses slowly, awareness of rare but potentially serious complications is crucial to improve outcomes.

The study highlights a need for further research into potential contributors, including the possibility that sicker patients may receive more prompt treatment.

Over the course of voxelotor treatment, hemoglobin levels and hemolysis markers improved, with results sustained over the treatment period.

The hesitation to prescribe nonsteroidal anti-inflammatory drugs (NSAIDs) to patients with hemophilia is not supported by current research, based on a recently published review, but the available studies had significant limitations.

Although the understanding of chronic graft-versus-host disease (GVHD) has improved in recent years, more work is needed to facilitate personalized treatments for patients after hematopoietic stem cell transplantation (HSCT).

The analysis emphasizes the association between vaso-occlusive crisis frequency and health care costs, which are inflated largely due to inpatient visits.

Gene therapy is a novel approach to hemophilia A treatment that carries a substantial cost up front but may lead to cost savings compared with current prophylaxis agents in the long run.

The findings suggest telenursing between in-person visits may help reduce distress related to symptoms in patients who have lung cancer.

A recent study supports the notion that non–driver mutations in essential thrombocythemia (ET) and polycythemia vera (PV) have predictive value and found that a proposed international prognostication model may be useful for Japanese patients.

This rare immune-related adverse event has only been previously reported 4 times in the context of immune checkpoint inhibitor (ICI) treatment.

A recently published report describes the diagnosis and treatment of a case of iron-deficiency anemia–associated thrombocytopenia secondary to recurrent nosebleeds.

Immune checkpoint inhibitors, either alone or in combination with chemotherapy, were associated with better outcomes in non–oncogene-addicted non–small cell lung cancer (NSCLC).

A study of women and girls from families with hemophilia in Thailand assessed the emotional impact of a carrier diagnosis and emphasized the value of comprehensive management.

JAK2-unmutated erythrocytosis has a variety of etiologies, and identifying the underlying cause may prove helpful in cases where it is secondary to another condition such as polycythemia vera.

Population-based data is a crucial aspect to evaluating current care quality and improving outcomes for patients with sickle cell disease.

Untreated bleeds often go undocumented during clinical trials, but reporting these bleeding events could provide additional insight into therapy efficacy.

Although no pharmacological treatment offers long-term efficacy or extends survival with Huntington disease at this point, research suggests that stem-cell therapy holds promise in this and other neurodegenerative diseases.

Despite the obstacles and risks, the review authors conclude that universal chimeric antigen receptor (CAR) T-cell therapy has the potential to play a key role and mitigate some of the limitations associated with autologous CAR T-cell therapy in cancer treatment.

A review published in the journal Immunotherapy summarizes the applications of sutimlimab and emphasizes the importance of individualizing treatment for patients with cold agglutinin disease.

A recent review summarized the current landscape of Bruton tyrosine kinase (BTK) inhibitors in both hematological cancers and inflammatory disorders.

The review concludes that CD20-targeted chimeric antigen receptor (CAR) T-cell therapy is an option worth exploring, despite potential safety concerns that warrant more extensive research.

The model was found to be predictive and may help identify patients with multiple myeloma at a high risk of early disease progression.

Within a selection of therapies showing efficacy over a placebo, lenalidomide-carfilzomib was a standout against the rest of the group.

Despite the use of therapies to address the clinical manifestations of myelofibrosis (MF) and essential thrombocythemia (ET), the symptom burdens of these conditions continue to negatively affect patient quality of life (QOL).

Findings of a recent study suggest that complications of sickle cell disease (SCD) significantly contribute to morbidity and are more prevalent among older patients.

A Swedish population-based study adds to data suggesting men have a higher risk of lymphoma and likely higher mortality.

The review highlighted key diagnostic and treatment considerations for patients with splanchnic vein thrombosis and myeloproliferative neoplasms.

An analysis of patient-reported outcomes from the pivotal CARDINAL trial of sutimlimab in cold agglutinin disease found that quality of life (QOL) improved and persisted throughout treatment.