“If only I had”, patients’ experiences during early oncology trials

IJsselt, Maaike E A van Sasse van; Biessen, Diane A.J. van der; Puffelen, Andrea J. van; Robbrecht, Debbie G.J.; Oldenmenger, Wendy H.

doi:10.1007/s00520-023-07738-y

Cited by 1 publication

(2 citation statements)

References 26 publications

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“…In fact, multiple surveys point to the fact that almost every clinician and patient volunteered for a phase 1 trial with the objective of a clinical response, 1,2 in addition to altruistic motive. 3 A review of published phase 1 trials reported ORRs ranging from 4% to 12.2%, [4][5][6][7][8][9][10][11] and CBRs ranging from 24% to 53%. [6][7][8][9][10][11] The selection of patients for phase 1 trials remains challenging.…”

Section: Introductionmentioning

confidence: 99%

“…However, secondary objectives, such as overall response rate (ORR; calculated as partial response [PR] plus complete response [CR]) and clinical benefit rate (CBR; calculated as stable disease plus ORR), are equally important end points. In fact, multiple surveys point to the fact that almost every clinician and patient volunteered for a phase 1 trial with the objective of a clinical response, in addition to altruistic motive . A review of published phase 1 trials reported ORRs ranging from 4% to 12.2%, and CBRs ranging from 24% to 53% …”

Section: Introductionmentioning

confidence: 99%

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Outcomes Among Racial and Ethnic Minority Patients With Advanced Cancers in Phase 1 Trials

Goel,

Negassa,

Ghalib

et al. 2024

JAMA Netw Open

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ImportancePatients from racial and ethnic minority groups (eg, Asian, Hispanic, and non-Hispanic Black patients) have low representation in clinical trials, especially in phase 1 trials in cancer. These trials represent valuable options for patients with advanced cancer who experience disease progression with standard therapy.ObjectiveTo determine whether the benefit of enrollment to phase 1 cancer trials extends to Asian, Hispanic, and non-Hispanic Black patients as much as it does for non-Hispanic White patients.Data SourcesPatient records at a single institution from January 1999 to December 2016 were reviewed. Treatment-related responses, toxic effects, and deaths were recorded.Study SelectionAll phase 1 studies were included.Data Extraction and SynthesisData underwent independent extraction by multiple observers following the Preferred Reporting Items for Systematic Reviews and Meta-analyses (PRISMA) reporting guideline.Main Outcomes and MeasuresThe primary outcome was overall survival (OS), assessed using univariate and multivariable time-to-event analyses.ResultsA total of 738 patients (median [range], 60 [22-93] years; 467 [63.3] female) including 197 Hispanic patients (26.7%), 238 non-Hispanic Black patients (32.2%), and 282 non-Hispanic White patients (38.2%), were enrolled in 64 phase 1 trials, including 33 cytotoxic trials (51.5%), 21 biologic trials (32.8%), and 10 combined therapy trials (15.6%). The primary cancer diagnoses were colorectal (187 patients [25.3%]), ovarian (141 patients [19.1%]), lung (58 patients [7.9%]), uterine (49 patients [6.6%]), and breast (41 patients [5.6%]). Patients underwent a median (range) of 3 (0-13) therapies prior to trial enrollment. Among 558 patients evaluated for response, the clinical benefit rate (ie, stable disease plus response rates) was 49.1%, and the overall response rate was 6.5%. Grade 3 or 4 nonhematological toxic effects were observed in 27.8% (95% CI, 24.6%-31.3%) of patients and grade 3 or 4 hematological toxic effects were observed in 19.7% (95% CI, 17.0%-22.8%) of patients. The treatment-related mortality rate was 0.9% (95% CI, 0.4%-1.9%). Median OS was 9.6 (95% CI, 8.2-11.0) months among Hispanic patients, 8.3 (95% CI, 6.7-10.4) months among non-Hispanic Black patients, and 9.8 (95% CI, 8.5-11.4) months among non-Hispanic White patients (P = .13). In a multivariable analysis, age older than 60 years, Eastern Cooperative Oncology Group performance status score of 2 or greater, more than 2 metastatic sites, lactate dehydrogenase grade 1 or 2, grade 2 or greater low albumin, grade 1 or greater total bilirubin, and grade 2 or greater anemia were associated with worse prognosis, whereas leukocytosis greater than grade 1 was associated with better OS.Conclusions and RelevanceIn this meta-analysis assessing outcomes in phase 1 cancer trials among patients from racial and ethnic minority groups, Hispanic and non-Hispanic Black patients had benefits similar to those of non-Hispanic White patients.

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