Validation of the Princess Margaret immune oncology prognostic index (PM-IPI) for patients (pts) treated in immune oncology (IO) early phase trials.

2017 ◽  
Vol 35 (15_suppl) ◽  
pp. 3070-3070
Author(s):  
Daphne Day ◽  
Anna Spreafico ◽  
Stephanie Lheureux ◽  
Albiruni Ryan Abdul Razak ◽  
Aaron Richard Hansen ◽  
...  
Keyword(s):  
Early Phase ◽  
Prognostic Score ◽  
Performance Status ◽  
Multivariable Analysis ◽  
Prognostic Index ◽  
Immune Checkpoints ◽  
Early Phase Trials ◽  
Metastatic Sites ◽  
Ecog Ps ◽  
Immune Oncology

3070 Background: We previously developed the PM-IPI (ECOG performance status [PS] > / = 1, albumin < lower limit of normal [LLN] and > 2 metastatic sites) from a retrospective cohort of 192 pts treated in phase I IO trials (development cohort). The PM-IPI prognosticated for overall survival (OS), 90-day mortality (90DM) and was associated with improved overall response rate (ORR) and progression free survival (PFS). Our aim was to prospectively validate the PM-IPI in an independent cohort of pts treated on IO trials. Methods: We included 152 consecutively treated advanced solid tumor pts at PM from Aug 2015 to Aug 2016 in 24 IO early phase trials, targeting immune checkpoints and/or co-stimulatory molecules. Pts from the development cohort were excluded. The ability of the PM-IPI to prognosticate OS and 90DM, and predict PFS and ORR was compared with the previously published Royal Marsden Hospital prognostic score (RMI: albumin < 35g/L, LDH > upper limit of normal and > 2 metastatic sites) using the C-index (0.5 = no discrimination, 1 = perfect discrimination) and Area Under the Curve (AUC). Results: Median age was 59y (range 20-86), 28%/72% of pts were ECOG PS 0/1, and 88% had at least 1 prior systemic therapy (range 0-7). The most common tumor sites were gastrointestinal (23%), gynecological (16%), head and neck (15%) and urological (10%). Median PFS and OS were 9.0 and 39.7 wk respectively and 90DM was 14%. ORR was 7% by RECIST 1.1, immune related RECIST or immune related response criteria. In multivariable analysis, ECOG PS > / = 1 (HR 2.7, p = 0.01), albumin < LLN (HR 2.1, p = 0.01) and > 2 metastatic sites (HR 1.8, p = 0.04) were independently prognostic for OS. Pts with a PM-IPI score of 2-3 compared to 0-1 had significantly shorter OS (HR 3.3, p < 0.0001), PFS (HR 1.7, p = 0.005) and higher 90DM (OR 12.2, p = 0.019), and a trend towards lower ORR (OR 0.4, p = 0.15). The prognostic performance of PM-IPI was superior to the RMI for OS and 90DM, but not PFS and ORR (Table). Conclusions: In this independent validation cohort, the PM-IPI prognosticated for OS and 90DM and was associated with PFS. Validation in a large external cohort is ongoing. [Table: see text]

scholarly journals Association of the Lung Immune Prognostic Index with Immunotherapy Outcomes in Mismatch Repair Deficient Tumors

Cancers ◽  
2021 ◽  
Vol 13 (15) ◽  
pp. 3776
Author(s):  
Edouard Auclin ◽  
Perrine Vuagnat ◽  
Cristina Smolenschi ◽  
Julien Taieb ◽  
Jorge Adeva ◽  
...  
Keyword(s):  
Checkpoint Inhibitors ◽  
Performance Status ◽  
Prognostic Index ◽  
Predictive Marker ◽  
Risk Groups ◽  
Two Factors ◽  
One Year ◽  
Metastatic Sites ◽  
Tumor Types

Background: MSI-H/dMMR is considered the first predictive marker of efficacy for immune checkpoint inhibitors (ICIs). However, around 39% of cases are refractory and additional biomarkers are needed. We explored the prognostic value of pretreatment LIPI in MSI-H/dMMR patients treated with ICIs, including identification of fast-progressors. Methods: A multicenter retrospective study of patients with metastatic MSI-H/dMMR tumors treated with ICIs between April 2014 and May 2019 was performed. LIPI was calculated based on dNLR > 3 and LDH > upper limit of normal. LIPI groups were good (zero factors), intermediate (one factor) and poor (two factors). The primary endpoint was overall survival (OS), including the fast-progressor rate (OS < 3 months). Results: A total of 151 patients were analyzed, mainly female (59%), with median age 64 years, performance status (PS) 0 (42%), and sporadic dMMR status (68%). ICIs were administered as first or second-line for 59%. The most frequent tumor types were gastrointestinal (66%) and gynecologic (22%). LIPI groups were good (47%), intermediate (43%), and poor (10%). The median follow-up was 32 months. One-year OS rates were 81.0%, 67.1%, and 21.4% for good, intermediate, and poor-risk groups (p <0.0001). After adjustment for tumor site, metastatic sites and PS, LIPI remained independently associated with OS (HR, poor-LIPI: 3.50, 95%CI: 1.46–8.40, p = 0.02. Overall, the fast-progressor rate was 16.0%, and 35.7% with poor-LIPI vs. 7.5% in the good-LIPI group (p = 0.02). Conclusions: LIPI identifies dMMR patients who do not benefit from ICI treatment, particularly fast-progressors. LIPI should be included as a stratification factor for future trials.

First-MIND: A phase Ib, open-label, randomized study to assess safety of tafasitamab (tafa) or tafa + lenalidomide (LEN) in addition to R-CHOP in patients with newly diagnosed DLBCL.

2021 ◽  
Vol 39 (15_suppl) ◽  
pp. 7540-7540
Author(s):  
David Belada ◽  
Katerina Kopeckova ◽  
Juan Miguel Bergua ◽  
Marc André ◽  
Ernesto Perez Persona ◽  
...  
Keyword(s):  
Performance Status ◽  
Randomized Study ◽  
International Prognostic Index ◽  
Prognostic Index ◽  
Dose Intensity ◽  
Newly Diagnosed ◽  
Open Label ◽  
Bulky Disease ◽  
Phase Ib ◽  
Ecog Ps

7540 Background: Tafasitamab is a humanized, Fc-modified anti-CD19 monoclonal antibody that enhances antibody-dependent cellular cytotoxicity and phagocytosis. It is FDA-approved with LEN for adult patients (pts) with relapsed/refractory (R/R) DLBCL ineligible for autologous stem cell transplantation. First-MIND (NCT04134936) is a Phase Ib, open-label, randomized study of tafa + R-CHOP or tafa + LEN + R-CHOP in newly diagnosed DLBCL. Methods: Eligible pts were ≥18 years, treatment-naïve, with histologically confirmed DLBCL not otherwise specified, international prognostic index (IPI) 2–5 and ECOG performance status (PS) 0–2. Pts with known double- or triple-hit and transformed lymphoma were excluded. Treatment (Tx) comprised six 21-day cycles of tafa (12 mg/kg IV, Day [D] 1, 8, 15) + R-CHOP (arm A) or tafa (12 mg/kg IV, D1, 8, 15) + LEN (25 mg orally, D1–10) + R-CHOP (arm B). G-CSF and VTE prophylaxis was mandatory. Primary objective is safety; secondary objectives are ORR, PET-CR rate at end of Tx, PFS, long-term safety, pharmacokinetics, immunogenicity. Results: From Dec 2019 to Aug 2020, 83 pts were screened in Europe and the US; 66 were randomized (33 per arm). Data cut-off for this analysis: 9 Dec 2020; study is ongoing. Median age was 64.5 years (range 20–86). Overall, 30% (20/66) of pts were ≥70 years and many had high-risk disease: IPI 2 29%, IPI 3 46%, IPI 4 26%. ECOG PS: 47% of pts were ECOG PS 0, 44% PS 1, 9% PS 2. Most pts had stage III/IV disease (92%); 46% had bulky disease. All pts experienced a treatment-emergent adverse event (TEAE). Grade ≥3 neutropenia and thrombocytopenia occurred in 54.5% and 12.1% (arm A) and 66.7% and 30.3% (arm B) of pts, respectively (Table). Serious TEAEs occurred in 42.4% (arm A) and 51.5% (arm B) of pts. There were three deaths, unrelated to tafa and/or LEN (sepsis, urosepsis, and COVID-19 pneumonia). R-CHOP dose intensity was maintained in both arms. Among 60 pts who completed tumor assessments after cycle 3, ORR was 89.7% (arm A) and 93.5% (arm B). Conclusions: These data suggest R-CHOP + tafa or tafa + LEN is tolerable in pts with Tx-naïve DLBCL and that R-CHOP dosing is not affected. Toxicities are similar to those expected with R-CHOP or R-CHOP + LEN. Updated safety and early efficacy data will be presented at the conference. Clinical trial information: NCT04134936. [Table: see text]

Predictors of outcome for pemetrexed (Pem) in metastatic NSCLC (mNSCLC).

2013 ◽  
Vol 31 (15_suppl) ◽  
pp. e19025-e19025
Author(s):  
Cortney Vanderbilt Jones ◽  
Lingling Du ◽  
Paul Elson ◽  
Tarek Mekhail ◽  
Nathan A. Pennell ◽  
...  
Keyword(s):  
Survival Data ◽  
Clinical Benefit ◽  
Performance Status ◽  
Single Agent ◽  
Multivariable Analysis ◽  
Cox Proportional Hazards Model ◽  
Front Line ◽  
Metastatic Nsclc ◽  
Poorly Differentiated ◽  
Metastatic Sites

e19025 Background: Pemetrexed (Pem) is the first agent showing different efficacy based on histology in the treatment (tx) of NSCLC. Our goal was to identify outcome predictors in patients (pts) with metastatic NSCLC (mNSCLC) treated with pem. Methods: Retrospective data of pts with mNSCLC who received pem were analyzed. Variables included demographics, ECOG performance status (PS), disease sites, pre/post-pem tx, and toxicities. Clinical benefit defined as complete response (CR), partial response (PR), and stable disease (SD) >6 months, progression-free survival (PFS), and overall survival (OS) were analyzed using logistic regression and Cox proportional hazards model. Results: 240 pts were included. 55% male, 84% smokers. 68% adenocarcinomas, 10% squamous/adenosquamous carcinomas. Median age 64 years (range 34-84). Pem was given for a median of 4 cycles (range 1-73), as 1st line in 20% and 2nd line in 50%, given as a single agent in 69%. The most common toxicities were constitutional (50%) and GI related (29%). 31% of pts achieved CR (n=4) or PR, 33% progressed, 36% had SD. Front-line pem use (p=.0003), adenocarcinoma histology (p=.05), and non-pulmonic metastatic sites ≤2 (p=.01) were independent predictors of clinical benefit, with response rate of 71% for pts with all three features, compared to only 7% for pts with none of these features. Multivariable analysis of survival data revealed ECOG PS >1, non-pulmonic metastatic sites>2, and squamous/poorly differentiated histology predicted poor PFS and OS. In addition, interval from diagnosis to start of pem <12 months and male gender predicted poor PFS, while former/current smoker predicted poor OS. Pts with favorable features had a median PFS of 7.7 months, compared to 2.1 months with unfavorable features (p<.0001). A median OS of 16 months was achieved in pts with favorable features, compared to 5.9 months with unfavorable features (p<.0001). Conclusions: Front-line pem use, adenocarcinoma histology, and ≤2 non-pulmonic metastatic sites predict better response to pem. Poor PS, >2 non-pulmonic metastatic sites, squamous/poorly differentiated histology predict poor PFS and OS. An analysis of biomarkers is ongoing.

Prognostic factors in metastatic gastric cancer patients (pts) treated with second-line chemotherapy

2007 ◽  
Vol 25 (18_suppl) ◽  
pp. 15169-15169
Author(s):  
V. Catalano ◽  
F. Graziano ◽  
D. Santini ◽  
A. M. Baldelli ◽  
P. Giordani ◽  
...  
Keyword(s):  
Gastric Cancer ◽  
Weight Loss ◽  
Prognostic Factors ◽  
Performance Status ◽  
Second Line ◽  
First Line ◽  
Second Line Chemotherapy ◽  
Metastatic Sites ◽  
Ecog Ps ◽  
Line Chemotherapy

15169 Background: Currently, there is no established second-line chemotherapy for pts with advanced gastric cancer who failed to respond or progressed after a first-line chemotherapy. Many of these pts still have a good performance status or have symptoms to be palliated at the time of first-line failure and are candidates for second-line chemotherapy. However, phase II trials demonstrate divergent results about pts more likely to benefit from second-line chemotherapy. We retrospectively analyzed the influence of various clinicopathologic factors on the survival of pts treated with second-line chemotherapy. Methods: Analysis is based on the data of 169 pts consecutively treated at 3 oncology department with a second-line chemotherapy. Univariate and multivariate analyses were performed to determine prognostic factors of survival. The variable used for analysis were: sex, age, ECOG performance status, a weight loss > 5 Kg in the last month; site of primary tumor, histopathology; hemoglobin, serum albumin, and CEA levels, number and site of metastatic disease, response to and time-to-progression (TTP) with the first- line chemotherapy. Results: The variables predictive of better survival were: ECOG PS 0–1 (p<0.001), no weight loss (p=0.001), hemoglobin level > 10 g/dl (p=0.01), CEA level <50 U/ml (p<0.02), number of metastatic sites = 2 (p=0.002), TTP of the first-line chemotherapy > 4 months (p=0.008). Peritoneal carcinomatosis was predictive of poor survival only when associated with one or more signs or symptoms as vomiting, anorexia, abdominal pain, ascites(p=0.03). Four factors were independently associated with better overall survival: ECOG PS 0–1 (p=0.002; HR 0.46; CI 95%, 0.29–0.75), CEA level <50 U/ml (p=0.008; HR 0.54; CI 95%, 0.35–0.85), one or two metastatic sites of disease (p=0.01; HR 0.58; CI 95%, 0.39–0.88), and TTP of the first-line chemotherapy > 4 months (p=0.02; HR 0.66; CI 95%, 0.45–0.95). Conclusions: In the absence of data deriving from randomized, controlled, clinical trials, this analysis suggests that some clinical factors may help clinicians to better select groups of pts with gastric cancer more likely to benefit from a second-line chemotherapy. No significant financial relationships to disclose.

Litmus test or destination systemic therapy? Trends in referral for surgery after initial systemic therapy in metastatic kidney cancer.

2017 ◽  
Vol 35 (6_suppl) ◽  
pp. 502-502 ◽  
Author(s):  
Liam Connor Macleod ◽  
Atreya Dash ◽  
George Schade ◽  
Scott S. Tykodi ◽  
John L. Gore
Keyword(s):  
Kidney Cancer ◽  
Systemic Therapy ◽  
Clinical Stage ◽  
Performance Status ◽  
Multivariable Analysis ◽  
Factors Associated ◽  
Analysis Survival ◽  
Litmus Test ◽  
Metastatic Sites ◽  
Systemic Therapies

502 Background: With nine new targeted and immunotherapeutic agents for metastatic kidney cancer (mRCC) since 2005, there is no randomized data supporting sequencing cytoreductive nephrectomy (CN) and newer systemic therapies (ST). Increased disease control with ST engenders concern that CN may shorten life or delay therapy. Thus, in all the best prognostic candidates, initial ST as a “litmus” test may be advocated prior to CN. We evaluated use of CN after initial ST, hypothesizing receipt of deferred CN to be associated with increased survival time, markers of increased performance status, less rapid disease, and socioeconomic status. Methods: The National Cancer Database was screened for adult patients with biopsy-proven mRCC treated with initial systemic therapy between 2006-2013. Covariates included demographic, oncologic, hospital-level, and geographic variables. Unadjusted and multivariable logistic regression was performed, identifying factors associated with CN after initial ST. Results: Of 14,651 patients treated with initial ST for mRCC, 709 (4.8%, median OS 19 months, IQR 9-35) underwent delayed CN compared with 13,942 (95.2%, median OS 5 months, IQR 2-13) treated with ST alone. On multivariable analysis, survival ≥3 months was highly associated with receipt of CN (OR 10.6, 95% CI 5.5, 20.5). However, of 9,796 surviving ≥3 months, only 689 (7%) underwent CN. Factors associated with lower odds of CN included older age, greater comorbidity, higher clinical stage (T and N), and unfavorable metastatic sites (i.e., brain, bone, liver) with all p<0.001. Educational attainment was associated with receipt of CN, but hospital characteristics and travel burden were not. Conclusions: Effectiveness of CN in the modern mRCC era is uncertain. Initial ST is typically for those with poor prognosis. Yet, after an initial litmus test, re-evaluation of risk rarely leads to CN. Socioeconomic factors may affect CN decision-making, a potential disparity that merits further investigation.

Prognostic score for second or further line immunotherapy in advanced non-small cell lung cancer (aNSCLC): An external validation.

2019 ◽  
Vol 37 (15_suppl) ◽  
pp. e14077-e14077
Author(s):  
Arsela Prelaj ◽  
Giuseppe Lo Russo ◽  
Claudia Proto ◽  
Diego Signorelli ◽  
Roberto Ferrara ◽  
...  
Keyword(s):  
Prognostic Score ◽  
Performance Status ◽  
External Validation ◽  
Progression Free Survival ◽  
Best Supportive Care ◽  
Second Line ◽  
Ecog Performance Status ◽  
Prognostic Groups ◽  
Recorded Data ◽  
Ecog Ps

e14077 Background: Beyond PD-L1, nowadays oncologists can only use clinical characteristics to candidate patients for immunotherapy (IO). Previously, a clinical prognostic score composed by ECOG performance status (PS), sex, histology, stage, uses of platin-based therapy at first-line (1L) and response to 1L categorized 3 different prognostic groups for patients treated with second-line (2L) chemotherapy (CHT) (Di Maio, EJC. 2010 Mar;46(4):735-43.). The aim of this study is to assess if the same score is able to discriminate the outcome of aNSCLC pts treated in 2L or further-line IO, potentially helping decision making. Methods: We recorded data of patients collected from two institutional databases: Istituto Nazionale Tumori of Milan and IRCCS Oncologico Giovanni Paolo II of Bari, Italy. Overall survival (OS) was the primary endpoint and also progression-free survival (PFS) was assessed. Prognostic score was generated, and pts were divided into 3 prognostic groups: best (B: < 5), intermediate (I:5-9), worst (W: > 9). Results: Overall, 347 pts were included in the analysis (193 from Milan and 154 from Bari). Median age was 66 years (y) (30 – 88y), most were < 70 y (67.5%), male (70.7%), smokers (79.5%) and adenocarcinoma (74.6%). ECOG PS was: 0 (23%), 1 (54.5%) and 2 (22.5%). Pts distribution was: 28%, 51% and 21% in the B, I and W groups, respectively. Median OS was 18.0 months for B group, 8.5 months for I group (HR vs B 1.83, 95%CI 1.35 – 2.47, p < 0.001) and 2.6 months for W group (HR vs B 5.77, 95%CI 3.99 – 8.33, p < 0.001). Median PFS was 3.4 months for B group, 3.7 months for I group (HR vs B 1.35, 95% CI 1.03 – 1.77, p = 0.032) and 1.9 months for W group (HR vs B 2.51. 95% CI 1.80- 3.50, p < 0.001). Similar results were obtained stratifying the model by Institution. Conclusions: This clinical prognostic score, that was generated in patients treated with second-line chemotherapy, is able to highly predict outcomes of patients treated with IO. These results demonstrated that in pre-treated aNSCLC pts, the worst category has a dismal absolute life expectancy, and probably would not benefit from any active systemic therapy (independently if CHT or IO). Perhaps for these pts best supportive care could be the best choice.

scholarly journals Outcome of patients with advanced endometrial and cervical cancer treated in a phase 1 unit.

2017 ◽  
Vol 35 (15_suppl) ◽  
pp. 5597-5597
Author(s):  
Rowan Miller ◽  
Michael-John Devlin ◽  
Nicholas Fraser Brown ◽  
Tami Grunewald ◽  
Mary McCormack ◽  
...  
Keyword(s):  
Early Phase ◽  
Clear Cell ◽  
Phase 1 ◽  
Performance Status ◽  
Treatment Options ◽  
Progression Free Survival ◽  
Histological Subtypes ◽  
Prior Therapy ◽  
Early Phase Trials ◽  
Specific Expansion

5597 Background: Patients (pts) with advanced endometrial (EC), cervical and vulval (CVC) cancer have limited therapeutic options and poor prognosis. Early phase trials may be a suitable option for pts with good performance status aided by molecular selection. We sought to determine the outcome of EC and CVC pts treated in a phase 1 unit. Methods: Medical records of pts with EC and CVC treated within an early phase trial between 2010 and 2016 were reviewed. Data comprised pt and tumor characteristics, prior therapy, trial therapy and outcome. Results: 38 pts, median age 59 years (21-74) with EC (19) or CVC (19) were identified. Median prior therapies for advanced disease: 1 (1-3). Histological subtypes: endometrioid (4), high grade serous (HGS 7), carcinosarcoma (CS 4), clear cell (1), and adenosquamous (3) for pts with EC; adenocarcinoma (5), squamous (11), clear cell (2) and neuroendocrine (1) for CVC pts. 20 pts (53%) had Next Generation Sequencing (NGS) using a targeted panel with actionable mutations identified in 10 (KRAS (4), PIK3CA (6) and EGFR (1)). Pts were allocated in order of priority to a trial (1) on the basis of NGS (‘genomic’ 8%), (2) within a ‘tumor specific’ expansion cohort (45%) or (3) a ‘generic’ study (47%). The overall response rate (ORR) was 21% with 34% stable disease (SD) and median progression free survival (PFS) and overall survival (OS) of 11 and 42 weeks respectively, with 10 pts still on study. Within the EC cohort ORR was 21% with 32% SD and PFS and OS of 9 and 38 weeks respectively. For the CVC cohort ORR was 21% with 37% SD and PFS and OS of 12 and 42 weeks respectively. Outcomes were better for the pts in the genomic and tumour specific groups. Both PFS and OS were longer with median PFS of 42, 32 and 8 weeks and OS of 91, not reached and 37 weeks for genomic, tumor specific and generic trials respectively. Conclusions: Early phase trials represent a good option for pts with advanced EC and CVC with meaningful clinical benefit observed even in this small cohort. Encouraging RR and PFS were observed in these pts with limited standard treatment options. This includes pts with difficult to treat histological subtypes such as HGS and CS EC and clear cell and adenocarcinoma CVC. NGS is feasible in real time and increasing use may benefit pts further.

scholarly journals Utility of the High-Sensitivity Modified Glasgow Prognostic Scores for Oropharyngeal Carcinoma

OTO Open ◽  
2021 ◽  
Vol 5 (3) ◽  
pp. 2473974X2110423
Author(s):  
Hiroyuki Iuchi ◽  
Junichiro Ohori ◽  
Yumi Ando ◽  
Takeshi Tokushige ◽  
Megumi Haraguchi ◽  
...  
Keyword(s):  
Prognostic Score ◽  
Performance Status ◽  
Univariate Analysis ◽  
Multivariable Analysis ◽  
Glasgow Prognostic Score ◽  
Disease Free Survival ◽  
High Sensitivity ◽  
Prognostic Scores ◽  
Albumin Concentration ◽  
Modified Glasgow Prognostic Score

Objective There is increasing evidence that the high-sensitivity modified Glasgow prognostic scores are inflammatory indices that can predict survival for many cancer types. However, there is limited information regarding their prognostic values in cases of head and neck cancer. This study aimed to evaluate whether the high-sensitivity modified Glasgow prognostic scores could predict outcomes among patients with oropharyngeal squamous cell carcinoma (OPC). Study Design Retrospective study. Setting University hospital. Methods We reviewed the records of 106 patients with histologically confirmed OPC between March 2009 and June 2020. The high-sensitivity modified Glasgow prognostic scores were calculated as 0 (C-reactive protein [CRP] concentration: ≤0.3 mg/dL), 1 (CRP concentration >0.3 mg/dL and albumin concentration ≥3.5 mg/dL), or 2 (CRP concentration >0.3 mg/dL and albumin concentration <3.5 mg/dL). Univariate and multivariable Cox proportional hazard analyses were performed for overall survival (OS) and disease-free survival (DFS). Results Forty-four of these patients had human papillomavirus (HPV)–positive OPC, and 62 had HPV-negative OPC, and these populations were analyzed separately. The high-sensitivity modified Glasgow prognostic score was significantly associated with age, performance status, and HPV. On univariate analysis, high-sensitivity modified Glasgow prognostic score showed associations with OS and DFS in both subpopulations. Moreover, on multivariable analysis, the high-sensitivity modified Glasgow prognostic score showed associations with OS and DFS in both subpopulations. Poor performance status predicted OS in both subpopulations. Conclusion We conclude that the high-sensitivity modified Glasgow prognostic score is useful as an independent prognostic factor in OPC.

scholarly journals Access to early phase clinical trials for children with relapsed and refractory neuroblastoma. A multicentre international study.

2021 ◽  
Author(s):  
Marta Cortes ◽  
Fernando Carceller ◽  
Alba Rubio-San-Simon ◽  
Sucheta Vaidya ◽  
Francisco Bautista ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Early Phase ◽  
Performance Status ◽  
International Study ◽  
Eligibility Criteria ◽  
Early Phase Trials ◽  
Novel Drugs ◽  
Innovative Therapies ◽  
Early Phase Clinical Trials ◽  
Relapsed Disease

Objectives. Neuroblastoma is the most common extracranial tumour in children, and prognosis for refractory and relapsed disease is still poor. Early Phase clinical trials play a pivotal role in the development of novel drugs. Ensuring adequate recruitment is crucial. The primary aim was to determine the rate of participation trials for children with refractory/relapsed neuroblastoma in two of the largest Drug Development European institutions. Methods. Data from patients diagnosed with refractory/relapsed neuroblastoma between January 2012 and December 2018 at the two institutions were collected and analysed. Results. Overall, 48 patients were included. A total of 31 (65%) refractory/relapsed cases were enrolled in early Phase trials. The main reasons for not participating in clinical trials included: not fulfilling eligibility criteria prior to consent (12/17, 70%) and screening failure (2/17, 12%). Median time on trial was 4.3 months (range 0.6-13.4). Most common cause for trial discontinuation was disease progression (67.7%). Median overall survival was longer in refractory (28 months, 95% CI, 20.9-40.2) than in relapsed patients (14 months, 95% CI, 8.1-20.1)) [p=0,034]. Conclusions. Although two thirds of children with refractory/relapsed neuroblastoma were enrolled in early Phase trials, recruitment rates can still be improved. The main cause for not participating on trials was not fulfilling eligibility criteria prior to consent, mainly due to performance status and short life expectancy. This study highlights the hurdles to access to innovative therapies for children with relapsed/refractory neuroblastomas and identifies key areas of development to improve recruitment to early phase trials.

Sign in / Sign up

Export Citation Format

Share Document