Haematopoietic gene therapy of non-conditioned patients with Fanconi anaemia-A: results from open-label phase 1/2 (FANCOLEN-1) and long-term clinical trials.

Background: Allogeneic haematopoietic stem-cell transplantation is the standard treatment for bone marrow failure (BMF) in patients with Fanconi anaemia, but transplantation-associated complications such as an increased incidence of subsequent cancer are frequent. The aim of this study was to evaluate the safety and efficacy of the infusion of autologous gene-corrected haematopoietic stem cells as an alternative therapy for these patients.

Methods: This was an open-label, investigator-initiated phase 1/2 clinical trial (FANCOLEN-1) and long-term follow-up trial (up to 7 years post-treatment) in Spain.

Recalling what we thought we knew about recall periods: a qualitative descriptive study of how adults diagnosed with cancer use recall periods for patient-reported outcome items about physical function.

Objectives: In cancer studies, assessment of patients' physical function can provide insight into cancer-related symptoms and the side effects of treatment. Physical function can be assessed using patient-reported outcome measures (PROMs), which may or may not include a recall period-the amount of time the questionnaire asks the respondent to think back to answer the questions. More understanding is needed about how patients interpret and respond to items posed with different recall periods, and which recall period they actually use.

Quality of end-of-life care in patients with hematological malignancies: potential role of palliative care.

Background: Limited data are available on the impact of specialist palliative care on end-of-life care quality for patients with hematologic malignancies. This study aims to analyze the quality of end-of-life care in patients with hematological malignancies, comparing those who receive palliative care with those who do not.

Methods: Observational, single center retrospective study.

Evaluation of Gemtuzumab Ozogamicin and Anthracycline Dosing for Favorable Risk Acute Myeloid Leukemia.

Gemtuzumab ozogamicin (GO) is a CD33-targeting antibody-drug conjugate approved for the treatment of CD33-positive de novo and relapsed and refractory acute myeloid leukemia (AML). Subset analyses have demonstrated improved clinical outcomes in patients with favorable-risk disease. It is unclear whether the addition of GO to cytarabine and anthracycline chemotherapy (7+3) improves clinical outcomes compared with other conventional regimens for AML.

Classic Hodgkin Lymphoma: The LYSA pragmatic guidelines.

Classic Hodgkin lymphoma (HL) is a distinct entity among hematological malignancies of B-cell origin. It is characterized by its unique histopathological features and generally favorable prognosis. Over the years, advancements in understanding its pathogenesis, coupled with refined diagnostic and evaluation modalities, as well as therapeutic strategies, have significantly transformed the landscape of HL management.

Patient, Care Partner, and Physician Voices in Treatment Decision-Making for Multiple Myeloma.

Introduction: Treatment decision-making for multiple myeloma (MM) is complex. Individuals involved in decision-making may value treatment attributes differently based on their role as a patient, care partner, or physician. This study describes those attributes, and what is most important by role.

Sustained remission at long term follow-up in adolescents and young adults with chronic primary immune thrombocytopenia.

Adolescents and young adults (AYAs) with immune thrombocytopenia (ITP) exhibit distinct clinical features and needs, defying categorization as either adults or children. Previous findings revealed a 50% risk of chronic disease at 12 months, yet the long-term course remains unclear. This study aimed to delineate the clinical and laboratory characteristics of AYAs with chronic primary ITP.

Perspectives on Childhood Lead Exposure Prevention: Looking Back and Looking Ahead.

Lead's neurotoxic properties and potential harmful effects to humans, particularly young children, have been recognized for decades, influencing public health policies to reduce its admixture in house paint and passenger car gasoline. We signal 3 emergent trends: firearm proliferation, complex international food supply chains, and equally complex product marketing strategies, which have opened opportunities for lead exposure to children from guns and ammunition, and lead contamination in children's food and consumer goods. Readers will also be apprised of Childhood Lead Poisoning Prevention Program and education strategies cultivated and advanced by the Centers for Disease Control and Prevention and its lead prevention partners.

The impact of language discordance on pediatric cancer care outcomes: A systematic review.

Language discordance between clinicians and families in pediatrics has been associated with adverse events and lower quality of care. We aimed to summarize the existing literature evaluating the impact of language discordance among healthcare professionals and families within pediatric oncology by conducting a systematic review. Of 8364 studies, 43 studies met eligibility for inclusion in this review.

Implementation of a Novel Pathway to Integrate Palliative and Oncology Care for Patients With Acute Myeloid Leukemia in a Community Hospital.

Purpose: Historically, patients with hematologic malignancies are referred to palliative care less often and later in the disease trajectory than those with solid tumors. Recent evidence demonstrates the benefit of early, integrated inpatient palliative care (PC) for patients with acute myeloid leukemia (AML) receiving chemotherapy at academic centers. The current study evaluated the feasibility of implementing standardized early palliative care services (PCS) during hospitalization for AML treatment in a community setting.

Machine Learning for Targeted Advance Care Planning in Cancer Patients: A Quality Improvement Study.

Context: Prognostication challenges contribute to delays in advance care planning (ACP) for patients with cancer near the end of life (EOL).

Objectives: Examine a quality improvement mortality prediction algorithm intervention's impact on ACP documentation and EOL care.

Methods: We implemented a validated mortality risk prediction machine learning model for solid malignancy patients admitted from the emergency department (ED) to a dedicated solid malignancy unit at Duke University Hospital.

A systematic review of health state utility values for older people with acute myeloid leukaemia.

Purpose: Older people with acute myeloid leukaemia (AML) have a poor prognosis, reduced health-related quality of life (HRQoL) and require substantial healthcare resources. The objectives of this systematic review were to determine what health state utility values (HSUVs) are reported in the literature that can be used in economic evaluations of interventions for older people with AML, identify research gaps, and discuss directions for future research.

Methods: The following databases were searched for studies published from inception until Feb 2023: PubMed, EMBASE, CINAHL, PsycINFO, Cochrane, and EconLit.

Top Ten Tips Palliative Care Clinicians Should Know About Caring for Patients with Myeloproliferative Neoplasms.

Myeloproliferative neoplasms (MPNs) are a group of rare chronic progressive blood cancers that vary widely in clinical presentation, yet all patients have a risk of disease progression and thrombotic complications. Diseases include primary myelofibrosis, polycythemia vera, and essential thrombocythemia. With current treatment approaches, most patients live a prolonged life, but many experience a complex of symptoms that negatively influence their functional status and quality of life.

Association of paediatric autoimmune cytopenia and inflammatory bowel disease suggests a common genetic origin.

The association of autoimmune cytopenia (AIC) and inflammatory bowel disease (IBD) has been reported in small series, but the incidence of and risk factors for IBD in children with AIC are not known. One thousand six hundred nine children with chronic immune thrombocytopenic purpura, autoimmune haemolytic anaemia or Evans syndrome from the prospective OBS'CEREVANCE cohort are included in this study. Overall, 15 children were diagnosed with IBD, including 14 who developed IBD after AIC diagnosis (median delay: 21 months).

Educating Communities, Families, and High School Students About Lead Exposure as a Public Health Problem.

The National Environmental Health Association strives to provide up-to-date and relevant information on environmental health and to build partnerships in the profession. In pursuit of these goals, we feature this column on environmental health services from the Centers for Disease Control and Prevention (CDC) in every issue of the . In these columns, authors from CDC's Water, Food, and Environmental Health Services Branch, as well as guest authors, will share tools, resources, and guidance for environmental health practitioners.

Timing of Palliative Care Consultation Impacts End of Life Care Outcomes in Metastatic Non-Small Cell Lung Cancer.

Context: Early specialist palliative care (PC) involvement in metastatic non-small cell lung cancer (mNSCLC) is associated with improved quality of life, less aggressive end of life (EoL) care, and longer survival. As treatment paradigms for NSCLC have evolved, PC utilization remains low.

Objectives: This work examines how the timing and extent of PC involvement impacts outcomes and the patient experience in mNSCLC in the era of immunotherapy.

Hodgkin lymphoma: hypodense lesions in mediastinal masses.

Hypodense volumes (HDV) in mediastinal masses can be visualized in a computed tomography scan in Hodgkin lymphoma. We analyzed staging CT scans of 1178 patients with mediastinal involvement from the EuroNet-PHL-C1 trial and explored correlations of HDV with patient characteristics, mediastinal tumor volume and progression-free survival. HDV occurred in 350 of 1178 patients (29.

How do patients interpret and respond to a novel patient-reported eastern cooperative oncology group performance status (ECOG)?

Purpose: Performance status is an important concept in oncology, but is typically clinician-reported. Efforts are underway to include patient-reported measures in cancer care, which may improve patient symptoms, quality of life and overall survival. The purpose of this study was to gain a preliminary understanding of how patients determined their physical performance status based on a novel patient-reported version of the Eastern Cooperative Oncology Group Performance Status (ECOG) scale.