In:
Journal of Clinical Oncology, American Society of Clinical Oncology (ASCO), Vol. 37, No. 15_suppl ( 2019-05-20), p. 2613-2613
Abstract:
2613 Background: Solid pediatric tumors that appear in adulthood are a heterogeneous group characterized by a low incidence, lack of standard therapeutic options and reduced survival. We have designed the first phase II clinical trial of nivolumab and ipilimumab in this setting, Here, we present the results of the first cohort with 30 evaluable patients. Methods: This is a multicenter, open-label, single arm Phase II study conducted in 15 centers of the Spanish Group for Rare Cancer (GETHI). We aimed to evaluate efficacy and safety of the combination of nivolumab and ipilimumab in adult patients ( 18 years) with locally advanced or metastatic childhood malignancies that have progressed or are not candidates to standard therapy. Treatment consisted on nivolumab 3 mg/kg IV q2w + ipilimumab 1 mg/kg IV q6w for 6 months or until progression/unacceptable toxicity, for a maximum of 24 months. Primary endpoint was overall response rate (ORR) according to RECIST v1.1 criteria. We used a Simon optimal two-stage design, with a first stage including first 30 evaluable patients. Results: 20 patients were male and median age was 43 (range 20-75). Most frequent histologies were medulloblastoma (4) neuroblastoma (4) and Ewing family tumors (3). 90% had received prior systemic therapy with 37% presenting progressive disease as best response. Median previous treatment lines were 3 (range 1-9). 27 patients were PS0-1, and 3 PS2. 6 patients have been treated for ≥6 months . Only one discontinued for adverse events. With a median follow up of 4,3 months (range 0,4-11,3), 1 patient has achieved a deep partial response (PR) (3,6%), 10 stable disease (SD) (35,7%) and 17 progressive disease (PD) (60,7%). 2 patients died before radiologic evaluation. Clinical benefit rate (CR+PR+SD) was 39,3%. Median progression free survival (PFS) was 1,8 months (95% CI 1,3-2,3), with a 3-months-PFS of 32,7% and 6-months-PFS of 20%. Median overall survival (OS) was 6,8 months (95% CI 3,3-10,2). 12 (40%) patients presented adverse events (AE) of any grade and 6 (20%) experienced a grade AE deemed as possibly related to treatment. Conclusions: The combination of nivolumab and ipilimumab showed significant clinical benefit in this population with little therapeutic options. One case of metastatic esthesioneuroblastoma, achieved a dramatic tumor response and represents the first patient with this extremely rare histology treated with immunotherapy. Clinical trial information: EudraCT 2016-003946-99.
Type of Medium:
Online Resource
ISSN:
0732-183X
,
1527-7755
DOI:
10.1200/JCO.2019.37.15_suppl.2613
Language:
English
Publisher:
American Society of Clinical Oncology (ASCO)
Publication Date:
2019
detail.hit.zdb_id:
2005181-5
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