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Patients with the rare disease typically present with hypertrophic cardiomyopathy and muscular hypotonia before the age of 1.

Findings are based on a retrospective, multicenter study carried out in Spain.

Newly presented data reveal disparities between Black and White patients in both the characteristics of and the decision-making around treatment for chronic lymphocytic leukemia (CLL) and multiple myeloma, respectively.

With the number of treatment options in hematology and oncology rapidly expanding, so too are the strategies for determining and ensuring cost-effectiveness, according to session speakers at the European Hematology Association (EHA) 2023 Congress.

Findings are based on a case-control study carried out in Brazil.

The first phase 3 results from the CARTITUDE-4 trial of ciltacabtagene autoleucel (cilta-cel) vs standard of care in lenalidomide-refractory multiple myeloma (MM) demonstrated the former’s convincing superiority in several key outcomes.

Results from the ASPEN and ALPINE trials of zanubrutinib showed greater improvements in quality of life (QOL) vs ibrutinib.

Jennifer Brown, MD, PhD, Dana-Farber Cancer Institute, shared her thoughts on the current research regarding Bruton tyrosine kinase (BTK) and B-cell lymphoma 2 (BCL-2) inhibitors in chronic lymphocytic leukemia (CLL) and what providers should keep in mind before prescribing them.

MD Anderson Cancer Center's Hans Lee, MD, shared considerations for possible infection risk associated with bispecific therapies used to treat relapsed/refractory multiple myeloma (MM) at the European Hematology Association 2023 Congress

An analysis of patients with metastatic castration-resistant prostate cancer (mCRPC) and homologous recombination repair (HRR) deficiency mutations showed that patients with BRCA mutations had poor outcomes compared with those without BRCA mutations and those with non-BRCA HRR mutations.

James F. Howard Jr, MD, professor of neurology at the University of North Carolina at Chapel Hill, explains how nerve and muscle signaling in the neuromuscular junction go awry in myasthenia gravis.

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

The pilot program for pulmonary arterial hypertension (PAH) was carried out at a single center in the Netherlands.

Tycel Phillips, MD, hematologist-oncologist at City of Hope in Duarte, CA, discussed the utility of epcoritamab in diffuse large B-cell lymphoma (DLBCL) and the potential impacts the bispecific antibody's recent approval will have in the community oncology setting.

High circulating tumor DNA (ctDNA) tumor fraction after induction chemotherapy identifies a population with an unmet need who has low chances to benefit from immune checkpoint blockades for non–small cell lung cancer (NSCLC), according to research discussed during a presentation at the American Society of Clinical Oncology Annual Meeting.

An abstract at the American Society of Clinical Oncology Annual Meeting showed disparities in lung cancer screening rates in an urban, multiethnic community, and patient navigation increased rates overall.

Lower socioeconomic status (SES) was not linked with hidradenitis suppurativa (HS) in minors, but was associated with obesity and tobacco use in this age group.

Authors concluded more research is needed to understand both patient level and structural barriers to inequitable access to multiple myeloma (MM) care in Canada.

The findings provide further support of recommendations in the clinical guidelines to favor one therapy over the other.

Investigators evaluated long-term outcomes among individuals who have eosinophilic esophagitis (EoE) and initiated first-line treatment with a food elimination diet after experiencing remission following stoppage of proton pump inhibitor monotherapy.

The autologous CAR T-cell therapy axicabtagene ciloleucel (axi-cel) demonstrated effectiveness and safety consistent with the ZUMA-5 trial when used in real-world settings to treat a broader population of patients with follicular lymphoma (FL).

Findings are based on a meta-analysis of 51 studies and 739 pregnancies.

Much work is needed to address inequities and gaps in care access for patients with acute myeloid leukemia (AML), said Jeffrey Lancet, MD, chair of the Department of Malignant Hematology at Moffitt Cancer Center.

Childhood myopia progression could be treated with low-dose atropine due to the safety and efficacy displayed in this study.

A friendly debate held at the European Hematology Association 2023 Congress focused on the potential for real-world data to improve the generalizability of clinical trial results but also acknowledged the limitations and current challenges with these data.

The Supreme Court protected Medicaid recipients’ right to sue nursing homes; wildfire smoke raises the risk of contracting COVID-19; CMS announced a new value-based primary care model.

Barb Kunz, MS, LCGC, senior genetic counselor at the US Oncology Network, discussed the importance of germline genetic testing in patients with triple-negative breast cancer and other cancer types.

Research presented at the American Society of Clinical Oncology Annual Meeting found prostate-specific membrane antigen ligand positron emission tomography (PSMA-PET) to be associated with worse overall survival in prostate cancer without distant metastasis based on conventional imaging.

If payers can agree on consensus criteria to improve care quality or costs, that will help physicians remember and try to be compliant, said Samyukta Mullangi, MD, MBA, incoming medical director at Thyme Care.

The use of immune therapies for hematological malignancies is expanding beyond chimeric antigen receptor (CAR) T-cell therapies in the traditional lymphoid disease states, but the impact on patient quality of life must remain top of mind, according to speakers at the European Hematology Association 2023 Congress.