scholarly journals Efficacy and safety of avelumab treatment in patients with metastatic Merkel cell carcinoma: experience from a global expanded access program

2020 ◽  
Vol 8 (1) ◽  
pp. e000313 ◽  
Author(s):  
John W Walker ◽  
Celeste Lebbé ◽  
Giovanni Grignani ◽  
Paul Nathan ◽  
Luc Dirix ◽  
...  
Keyword(s):  
Cell Carcinoma ◽  
Real World ◽  
Progressive Disease ◽  
Complete Response ◽  
Immunocompromised Patients ◽  
Merkel Cell ◽  
Efficacy And Safety ◽  
Expanded Access ◽  
Expanded Access Program ◽  
Access Program

BackgroundAvelumab, a human anti–programmed death-ligand 1 immunoglobulin G1 monoclonal antibody, showed favorable efficacy and safety in patients with metastatic Merkel cell carcinoma (mMCC) in the phase II JAVELIN Merkel 200 trial, leading to approval in multiple countries. We describe real-world experience with avelumab in patients with mMCC from an expanded access program.MethodsEligible patients had mMCC and progressive disease during or after chemotherapy or were ineligible for chemotherapy or clinical trial participation. Patients received an initial 3-month supply of avelumab (administered as 10 mg/kg intravenously every 2 weeks until progressive disease or unacceptable toxicity); resupply was allowed following complete response, partial response, stable disease, or clinical benefit per physician assessment.ResultsBetween December 15, 2015, and March 4, 2019, 558 of 620 requests from 38 countries were medically approved, and 494 patients received avelumab. Among 240 evaluable patients, the objective response rate was 46.7% (complete response in 22.9%, including 3 of 16 potentially immunocompromised patients), and the disease control rate was 71.2%. The median duration of treatment in evaluable patients with response was 7.9 months (range, 1.0–41.7) overall and 5.2 months (range, 3.0–13.9) in immunocompromised patients. No new safety signals were identified. The expanded access program closed for new requests on December 31, 2018, as required after regulatory approval; benefitting patients continued to receive avelumab.ConclusionsThe avelumab expanded access program for patients with mMCC demonstrated efficacy and safety in a real-world setting, consistent with the results from JAVELIN Merkel 200, and provided a treatment for patients with limited options.

scholarly journals Avelumab treatment in Italian patients with metastatic Merkel cell carcinoma: experience from an expanded access program

2021 ◽  
Vol 19 (1) ◽  
Author(s):  
Giovanni Grignani ◽  
Vanna Chiarion Sileni ◽  
Carmine Pinto ◽  
Roberta Depenni ◽  
Nicola Fazio ◽  
...  
Keyword(s):  
Partial Response ◽  
Cell Carcinoma ◽  
Merkel Cell Carcinoma ◽  
Objective Response ◽  
Complete Response ◽  
Trial Participation ◽  
Merkel Cell ◽  
Expanded Access ◽  
Expanded Access Program ◽  
Access Program

Abstract Background The incidence of Merkel cell carcinoma (MCC), a rare form of skin cancer with a poor prognosis, has increased in Italy in recent decades. Avelumab, an anti-programmed death ligand 1 monoclonal antibody, is approved for the treatment of metastatic MCC (mMCC) based on the results of the phase 2 JAVELIN Merkel 200 trial. The global avelumab expanded access program (EAP) was designed to provide compassionate use of avelumab prior to approval for patients with mMCC who had limited treatment options. We report findings from a subgroup of Italian patients enrolled in the avelumab EAP. Methods Eligible patients had mMCC and progressive disease following ≥ 1 prior line of chemotherapy or were ineligible for chemotherapy or clinical trial participation. Patients received avelumab 10 mg/kg intravenously every 2 weeks. Treating physicians were provided with an initial 3-month supply of avelumab; resupply was permitted if the patient achieved a complete response, partial response, stable disease, or other clinical benefit per physician assessment. Safety and efficacy data for the EAP were reported at the treating physician’s discretion. Results Between April 1, 2016, and September 14, 2018, 109 requests for avelumab were received from Italy, and 102 were approved. All but 1 of the approved patients had received ≥ 1 prior line of therapy. At data cutoff (March 22, 2019), 95 patients had been supplied with avelumab and response data were available for 55 patients. The objective response rate in response-evaluable patients was 29.1%, including 6 patients (10.9%) who achieved a complete response and 10 patients (18.2%) who achieved a partial response; in the total population supplied with avelumab (n = 95), the proportion who had an objective response was 16.8%. The median duration of treatment in responding patients was 9.7 months (range, 3.5–41.7 months). The most frequently reported treatment-related adverse events were infusion-related reaction (single preferred term; n = 3 [3.2%]) and pyrexia (n = 2 [2.1%]). Conclusions Results from Italian patients enrolled in the avelumab EAP are consistent with the findings of the JAVELIN Merkel 200 trial and confirm the efficacy and safety of avelumab treatment in this population.

scholarly journals Avelumab in European patients (pts) with metastatic Merkel cell carcinoma (mMCC): Experience from an ad-hoc expanded access program (EAP)

2018 ◽  
Vol 29 ◽  
pp. viii460-viii461
Author(s):  
P. Nathan ◽  
V. Kasturi ◽  
L. Dirix ◽  
E. Fenig ◽  
P.A. Ascierto ◽  
...  
Keyword(s):  
Cell Carcinoma ◽  
Merkel Cell Carcinoma ◽  
Ad Hoc ◽  
Merkel Cell ◽  
Expanded Access ◽  
Expanded Access Program ◽  
Access Program

REACT expanded-access program in patients with metastatic renal cell carcinoma: real-world data from a European subanalysis

2015 ◽  
Vol 11 (21) ◽  
pp. 2893-2903 ◽  
Author(s):  
Sergio Bracarda ◽  
Sylvie Rottey ◽  
Amit Bahl ◽  
Christian Eichelberg ◽  
Begoña Mellado ◽  
...  
Keyword(s):  
Renal Cell Carcinoma ◽  
Cell Carcinoma ◽  
Metastatic Renal Cell Carcinoma ◽  
Real World ◽  
Renal Cell ◽  
Real World Data ◽  
World Data ◽  
Expanded Access ◽  
Expanded Access Program ◽  
Access Program

scholarly journals Erdafitinib treatment in Brazilian patients with metastatic urothelial carcinoma (mUC): real-world evidence from an Expanded Access Program

2021 ◽  
Vol 13 ◽  
pp. 175883592110154
Author(s):  
Fernando Sabino M. Monteiro ◽  
Adriano Gonçalves e Silva ◽  
Andrea Juliana P. de S. Gomes ◽  
Carolina Dutra ◽  
Naira Oliveira Ferreira ◽  
...  
Keyword(s):  
Urothelial Carcinoma ◽  
Real World ◽  
Objective Response ◽  
Significant Activity ◽  
Efficacy And Safety ◽  
Expanded Access ◽  
Expanded Access Program ◽  
Real World Evidence ◽  
Metastatic Urothelial Carcinoma ◽  
Access Program

Background: Erdafitinib is the first targeted therapy approved for the treatment of patients with metastatic urothelial carcinoma (mUC). Approval was based on a phase II single-arm trial that demonstrated significant activity of erdafitinib in patients with tumors harboring FGFR2/3 alterations. In Brazil, an Expanded Access Program (EAP) provided patients with early access to erdafitinib prior to market authorization. The current report describes characteristics and outcomes of patients with mUC on erdafitinib therapy. Methods: Patients with mUC that failed first- and second-line systemic therapies were screened for FGFR2/3 alterations in primary or metastatic tumor tissues. Patients with FGFR2/3 alterations were selected to receive erdafitinib at the standard dosing schedule and were followed prospectively to evaluate the efficacy and safety outcomes. Results: From 19 April 2019, through 13 March 2020, 47 patients with mUC from 10 Brazilian centers were tested for FGFR2/3 alterations. Alterations in FGFR2/3 were found in 12 patients (25.5%) and all of them were eligible for the EAP. Four patients (33%) had partial response, while two patients (17%) had stable disease. Progressive disease, the best response, was observed in five patients (42%). At a median follow-up of 16.2 months, the median time to treatment failure (TTF) was 2.8 months. When considering only patients with objective response, the median TTF was 5.3 months. Adverse events (AEs) were reported for any grade and grade 3 or higher in 10 patients (83%) and 5 patients (42%), respectively. The most common AE was hyperphosphatemia. Conclusion: This first real-world evidence report of heavily treated patients with mUC confirms the efficacy and safety of erdafitinib in a disease setting with a lack of treatment options.

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