Journal of the National Comprehensive Cancer Network : JNCCN最新文献

Background: Only 3%-5% of patients with cancer nationwide enroll in clinical trials. Although participation among patients with solid tumors has been well studied, data in the hematopoietic stem cell transplantation (HSCT) setting remain limited. We sought to (1) describe clinical trial participation rates by HSCT type; (2) examine associations between patient, disease, and transplant characteristics and trial participation; and (3) explore the association between trial participation and overall survival (OS).

Methods: We conducted a retrospective study using Center for International Blood and Marrow Transplant Research (CIBMTR) data from US adults aged 18-75 years who underwent autologous or allogeneic HSCT between 2013 and 2019 (N=84,151; autologous: n=51,133; allogeneic: n=33,018). Logistic regression models assessed predictors of participation, while Cox models evaluated associations with OS.

Results: Trial participation was 2.0% among patients undergoing autologous HSCT and 11.1% among those undergoing allogeneic HSCT. Among autologous recipients, greater comorbidity burden was associated with lower odds of participation. Patients with multiple myeloma had higher odds than those with non-Hodgkin lymphoma (odds ratio [OR], 1.25; P=.01). Among allogeneic recipients, older adults had higher odds of participation (eg, OR, 2.19 for age ≥70 vs 18-19 years; P<.0001), as did non-Hispanic Black patients compared with non-Hispanic White patients (OR, 1.35; P=.0002). Participation was higher with cord blood grafts, mismatched unrelated donors, and certain diagnoses (eg, sickle cell disease). Lower participation was observed for severe aplastic anemia and haploidentical or peripheral blood grafts. Trial participation was not associated with OS in either group.

Conclusions: Clinical trial participation remains low, particularly among autologous HSCT recipients, younger adults, and patients with specific diagnoses. Findings highlight the need to broaden trial eligibility, enhance outreach, and ensure equity in trial access to promote generalizable, patient-centered evidence in HSCT.

Background: Patients with cancer often require hospital-level care to address urgent needs and symptom concerns. However, little research has sought to identify patient factors, particularly rural-urban residence, associated with care outcomes in this population.

Methods: We conducted a retrospective cohort study including adult patients with cancer admitted to the University of Oklahoma Medical Center from January 2017 through December 2022. We extracted patients' sociodemographic (ie, age, sex, race, ethnicity, rural-urban residence [Rural-Urban Continuum Codes]) and clinical (ie, Charlson comorbidity index [CCI] score, cancer type, metastatic disease status) information from the electronic health record. We used multivariable models to explore associations of patient factors with clinical outcomes (time to hospital discharge for length of stay [LOS], time to first readmission, and time to death [overall survival]).

Results: We identified 20,683 patients in our cohort (mean [SD] age, 62.6 [15.4] years; 52% female sex, 40% with a CCI score ≥3, 41% with metastatic cancer, 31% with nonmetropolitan residence). Median hospital LOS was 4.0 days, with 30- and 90-day readmission rates of 15% and 23%, and median survival was 5.0 years. Older age (hazard ratio [HR], 0.93; P<.001), male sex (HR, 0.94; P<.001), higher comorbidity (CCI score ≥3: HR, 0.70; P<.001), and metastatic disease (HR, 0.88; P<.001) were associated with longer LOS. Higher comorbidity (CCI score ≥3: HR, 2.70; P<.001) was associated with a greater risk of readmission. Older age (HR, 1.50; P<.001), CCI score ≥3 (HR, 1.68; P<.001), and nonmetropolitan residence (HR, 1.08; P=.007) were associated with worse survival.

Conclusions: In this cohort of hospitalized patients with cancer, we identified associations of patients' sociodemographic and clinical factors with their hospital LOS, risk of readmission, and survival. Findings from this work could help inform targeted efforts to enhance care delivery and outcomes for this population.

Background: Metastatic sarcomatoid renal cell carcinoma (msRCC) is an aggressive disease typically associated with poor outcomes following VEGF-targeted therapies. This study evaluated in real-world outcomes in patients with msRCC based on whether they received treatment with immune checkpoint inhibitors (ICIs).

Methods: We conducted a multi-institutional, retrospective study of patients who received systemic therapy for msRCC between July 2010 and June 2020. In total, 203 patients across 6 US academic centers were included. Patients were categorized into 2 groups based on whether they received ICIs: ICI+ (n=155) and ICI- (n=48). Overall survival (OS) was estimated using the Kaplan-Meier method and measured from the initiation of frontline systemic therapy. Treatment responses were evaluated by investigators at participating centers.

Results: After a median follow-up of 48.1 months, OS was significantly longer in the ICI+ group (median OS, 31.0 vs 7.6 months; hazard ratio [HR], 0.38; 95% CI, 0.26-0.56; P<.001). ICI+ status (HR, 0.38; 95% CI, 0.25-0.59; P<.001), non-clear cell or unclassified histology (HR, 2.99; 95% CI, 1.86-4.79; P<.001), and sarcomatoid component ≥25% (HR, 1.69; 95% CI, 1.11-2.59; P=.015) were independently predictive of OS. OS benefit in ICI+ group was maintained in the second line or later (median OS, 39.6 vs 7.6 months; HR, 0.32; 95% CI, 0.21-0.49; P<.001). Among patients with non-clear cell or unclassified histology, the ICI+ group had longer OS (median, 18.1 vs 7.0 months; HR, 0.20; 95% CI, 0.09-0.44; P<.001). Objective (39.4% vs 18.8%; P=.014) and complete (11.0% vs 4.2%; P=.258) response rates were higher in the ICI+ group.

Conclusions: A potential benefit of ICI-based therapy is suggested in both clear cell and non-clear cell msRCC. The benefit was observed in both the first-line setting and in second-line or later lines of therapy.

Background: Although Wilms tumor (WT) is the most common kidney cancer of childhood, it also presents infrequently in adolescents and even adults. Older patients with WT have inferior survival, but the reasons for this are unclear, and age is not currently used for therapeutic risk stratification. This study evaluates age as a prognostic variable and examines the influence of clinical and biological features on outcomes in a cohort of older patients with WT.

Patients and methods: We evaluated 4,100 patients with unilateral WT on Children's Oncology Group protocol AREN03B2. "Standard-age" patients (age 0-9 years) were compared with those presenting at "older-age" (age 10-30 years). The impact of age on survival was evaluated across the entire cohort and stratified by stage and histology.

Results: There were 3,936 standard-age and 164 older-age patients. Older-age patients were more likely to have higher local (P=.009) and overall disease stage (P<.001), larger tumor diameter (P<.001), longer intervals between diagnostic imaging and renal surgery (P<.001), no lymph nodes sampled (P<.001), and treatment that included radiation (P=.010). No older-age patients had WT predisposition syndromes. The effect of age as a continuous variable was significantly associated with worse prognosis for event-free and overall survival after accounting for differences in disease stage and histology. There was neither higher rates of anaplasia nor high-risk tumor cytogenetics in older-age patients to account for this survival disadvantage.

Conclusions: Advancing age is an inferior continuous prognostic risk factor in patients with unilateral WT, independent of conventional risk stratification parameters of stage and histology. Future studies should prospectively interrogate the biology of older-age patients and incorporate advanced age into therapeutic risk stratification.

Background: This qualitative study sought to understand factors related to low rates of screening mammography among transgender and gender-diverse (TGD) individuals and how these rates might be improved.

Methods: Individuals who self-reported as TGD were recruited from multiple sites and national patient advocates. Consenting participants completed an individual interview using a semistructured guide informed by the Health Belief Model (HBM). Interview data were analyzed using thematic analysis.

Results: A total of 44 individuals completed the interview (median age, 44.9 years; range, 19-77 years); 11% were a racial/ethnic minority; and 52%, 20%, and 25%, respectively, self-identified as a transgender woman, transgender man, or nonbinary. Three themes emerged around constructs in the HBM. First, individuals voiced a hope to be treated equally ("I just want to go to work, pay my taxes, love my family, and go along my day and be a good human") and described how such equitable treatment during screening guarded against gender dysphoria and favored gender affirmation ("it is affirming…being treated as any other woman"). Second, barriers and facilitators related to mammography were identified, including the need to address cost/low cost ("screenings are a luxury"); prior negative/positive experiences ("they would call my name and look at me but dismiss me"); absence/presence of education ("none of the doctors…ever told me what that process looks like"); and perceived low/high cancer risk ("my mom died pretty young from breast cancer"). Finally, practical recommendations were offered, including individual changing/waiting rooms, hiring gender-diverse staff, and engaging clinicians who provide whole-person, trauma-informed care.

Conclusions: Mammographic screening among TGD individuals could be improved by proactively creating safe, affirming spaces; working to lower costs; providing educational materials tailored to the TGD community about cancer risk and the practical aspects of screening; and hiring gender-diverse staff. These recommendations from TGD participants merit implementation as well as further study.

Background: Patients with stage IIIA cutaneous melanoma have relatively favorable prognoses (5-and 10-year overall survival probabilities of 93% and 88%, respectively), yet they are considered for systemic adjuvant therapy. Identifying the subset of patients at highest risk of recurrence could limit adjuvant treatment-related toxicity and direct therapy to those most likely to benefit.

Patients and methods: Patients aged ≥18 years with stage IIIA melanoma diagnosed between 2001 and 2016 at 36 centers, primarily in Europe and North America, were included in the study. The full cohort was split in a 3:1 ratio into development (n=1,386) and validation (n=448) cohorts. A random forest procedure was applied to the development cohort to select variables for inclusion. The selected variables were incorporated into a Fine-Gray model to predict recurrence-free survival and graphically represented as a nomogram. Model performance was assessed using the C-index and inspection of the calibration plot. The model's clinical utility was assessed using decision curve analysis and compared with alternative strategies.

Results: Eight variables were included in the model and were associated with the incidence of recurrence: mitotic rate (P<.001), number of positive sentinel lymph nodes (SLNs) per nodal basin (P<.001), diameter of the largest focus in the SLN (P=.001), ulceration (P<.001), age (P=.022), number of metastatic foci in the SLN (P=.019), lymphovascular invasion (P<.001), and extranodal extension (P=.005). In the validation cohort, the model had a C-index of 80.9% and appeared to be well calibrated. Decision curve analysis demonstrated a significant net benefit for adjuvant therapy guided by the nomogram compared with alternative strategies, including treating all patients with adjuvant therapy, treating no patients, or treating only patients with stage IIIA disease and >1.0-mm nodal tumor burden.

Conclusions: Our prediction model can improve patient selection for adjuvant therapy compared with other available strategies.

Hepatocellular carcinoma (HCC) is the most common primary cancer of the liver. The NCCN Guidelines for HCC provide recommendations for the comprehensive care of patients with HCC, including screening, evaluation, treatment, and surveillance. The multidisciplinary panel of experts meets at least once a year to discuss updates to the NCCN Guidelines based on new and emerging data. These Guidelines Insights focus on some of the recent updates.

The NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines) for Pancreatic Adenocarcinoma provide multidisciplinary diagnostic workup, staging, and treatment recommendations for resectable, borderline resectable, unresectable, locally advanced, and metastatic disease. This article summarizes the studies and Panel discussion that serve as the rationale for comprehensive care recommendations included in the NCCN Guidelines for Pancreatic Adenocarcinoma.