Children with cancer who receive PC experience a high burden of intensive treatments and often die in inpatient intensive care settings. Delayed PC involvement is associated with increased odds of dying in the intensive care unit. Prospective investigation of early PC involvement in children with high-risk cancer is needed to better understand potential impacts on cost-effectiveness, quality of life, and delivery of goal concordant care.

Hematologic malignancy, cancer-directed therapy at the end of life, and delayed documentation of advance directives are associated with late PC involvement in children who died of cancer. Identification of these variables affords opportunities to study targeted interventions to enhance access to earlier PC resources and services for children with high-risk cancer and their families.

The phenotype of spinal muscular atrophy (SMA) has been changing with the recent availability of three FDA‐approved treatments: intrathecal nusinersen, intravenous onasemnogene abeparvovec‐xioi, and enteral risdiplam. The degree of improvement in muscle strength and respiratory health varies with SMA genotype, severity of baseline neuromuscular and pulmonary impairment, medication used, and timing of the first dose. A spectrum of pulmonary outcomes has been reported with these novel medications when used early and in conjunction with proactive multidisciplinary management of comorbidities. In this review, we summarize the reported impact of these novel therapies on pulmonary well‐being and the improving trajectory of pulmonary morbidity, compared to the natural history of SMA. The importance of ongoing clinical monitoring albeit the improved phenotype is reiterated. We also discuss the limitations of the current SMA‐therapy trials and offer suggestions for future clinical‐outcome studies and long‐term monitoring.

Children with cancer who die in high-acuity inpatient settings often experience a high burden of intensive therapy at the end of life. Strategies to identify patients at higher risk of dying in the pediatric intensive care unit (PICU) have not been explored previously. This study finds that certain variables may predict PICU death for pediatric palliative oncology patients, including delayed palliative care involvement. Preemptive identification of patients at risk for PICU death affords opportunities to study the effects of earlier palliative care integration and increased discussions around preferred location of death on end-of-life outcomes for children with cancer and their families.

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Background: Racial and ethnic disparities in the provision of end-of-life care are well described in the adult oncology literature. However, the impact of racial and ethnic disparities at end of life in the context of pediatric oncology remains poorly understood. Objective: To investigate associations between end-of-life experiences and race/ethnicity for pediatric patients with cancer. Methods: A retrospective cohort study was conducted on 321 children with cancer enrolled on a palliative care service at an urban pediatric cancer who died between 2011 and 2015. Results: Compared to white patients, black patients were more likely to receive cardiopulmonary resuscitation (CPR; odds ratio [OR]: 4.109, confidence interval [CI]: 1.432-11.790, P = .009) and underwent 3.136 (CI: 1.433-6.869, P = .004) CPR events for every 1 white patient CPR event. The remainder of variables related to treatment and end-of-life care were not significantly correlated with race. Hispanic patients were less likely to receive cancer-directed therapy within 28 days prior to death (OR: 0.493, CI: 0.247-0.982, P = .044) as compared to non-Hispanic patients, yet they were more likely to report a goal of cure over comfort as compared to non-Hispanic patients (OR: 3.094, CI: 1.043-9.174, P = .042). The remainder of variables were not found to be significantly correlated with ethnicity. Conclusions: Race and ethnicity influenced select end-of-life variables for pediatric palliative oncology patients treated at a large urban pediatric cancer center. Further multicenter investigation is needed to ascertain the impact of racial/ethnic disparities on end-of-life experiences of children with cancer.

Background Cystic fibrosis (CF) is a multisystem disease with progressive deterioration. Recently, CF transmembrane conductance regulator (CFTR) modulator therapies were introduced that repair underlying protein defects. Objective of this study was to determine the impact of elexacaftor–tezacaftor–ivacaftor (ETI) on clinical parameters and inflammatory responses in people with CF (pwCF). Methods Lung function (FEV1), body mass index (BMI) and microbiologic data were collected at initiation and 3‐month intervals for 1 year. Blood was analyzed at baseline and 6 months for cytokines and immune cell populations via flow cytometry and compared to non‐CF controls. Results Sample size was 48 pwCF, 28 (58.3%) males with a mean age of 28.8 ± 10.7 years. Significant increases in %predicted FEV1 and BMI were observed through 6 months of ETI therapy with no change thereafter. Changes in FEV1 and BMI at 3 months were significantly correlated (r = 57.2, p < 0.01). There were significant reductions in Pseudomonas and Staphylococcus positivity (percent of total samples) in pwCF through 12 months of ETI treatment. Healthy controls (n = 20) had significantly lower levels of circulating neutrophils, interleukin (IL)‐6, IL‐8, and IL‐17A and higher levels of IL‐13 compared to pwCF at baseline (n = 48). After 6 months of ETI, pwCF had significant decreases in IL‐8, IL‐6, and IL‐17A levels and normalization of peripheral blood immune cell composition. Conclusions In pwCF, ETI significantly improved clinical outcomes, reduced systemic pro‐inflammatory cytokines, and restored circulating immune cell composition after 6 months of therapy.

Background In people with cystic fibrosis (pwCF), the impact of cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapies, such as Elexacaftor–Tezacaftor–Ivacaftor (ETI), on structural changes in the lungs is unclear. Objective To determine the impact of ETI on clinical parameters and on structural lung disease as measured by the changes in the chest computed tomography (CT) scans in pwCF. Methods Percent predicted forced expiratory volume in one second (ppFEV1), body mass index (BMI), and microbiologic data were collected at initiation and 3‐month intervals for 1 year. Chest CT scans before starting ETI therapy (baseline) and at 1‐year on ETI therapy were compared by two pulmonologists independently. Results The sample size was 67 pwCF, 30 (44.8%) males, median age of 25 (16, 33.5) years. Significant increases in ppFEV1 and BMI observed by 3 months of ETI therapy persisted throughout 1 year of ETI therapy (p < 0.001 at all‐time points for both). After 1 year on ETI, pwCF had significant reductions in Pseudomonas aeruginosa (−42%) and MRSA (−42%) positivity. None of the pwCF had worsening of chest CT parameters during 1 year of ETI therapy. Comparing chest CT findings at baseline and at 1‐year follow‐up, bronchiectasis was present in 65 (97%) pwCF and at 1‐year follow‐up decreased in 7 (11%). Bronchial wall thickening 64 (97%), decreased in 53 (79%). Mucous plugging in 63 (96%), absent in 11 (17%), and decreased in 50 (77%). Hyperinflation/air trapping in 44 (67%), decreased in 11 (18%), absent in 27 (44%) Conclusions ETI significantly improved clinical outcomes and lung disease as documented by improvement in chest CT scans.