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Clinical outcomes and survival of patients with myeloma and lymphoma enrolled into phase I clinical trials
Author(s) -
ElSharkawi Dima,
Ng ChinHin,
Payne Elspeth M.,
Yong Kwee L.,
Ardeshna Kirit M.,
Khwaja Asim,
Townsend William,
Popat Rakesh
Publication year - 2019
Publication title -
british journal of haematology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.907
H-Index - 186
eISSN - 1365-2141
pISSN - 0007-1048
DOI - 10.1111/bjh.15457
Subject(s) - medicine , clinical trial , common terminology criteria for adverse events , tolerability , adverse effect , multiple myeloma , hematology , oncology
Phase I clinical trials are primarily designed to assess the safety and toxicity of a new agent and determine the recommended dose for further trials that assess efficacy. Patients entering these trials typically have multiply relapsed or refractory disease and would otherwise receive supportive treatment. The collective outcomes of patients enrolled onto phase I trials in solid tumors but not in hematological malignancies have previously been reported. We analyzed the overall outcomes and survival of 91 patients enrolled onto one of 14 Phase I and I/II trials for hematological malignancies at a dedicated early phase trials unit within a tertiary hematology unit. Median time on trial was 3.7 months (range 0-24), 32% experienced grade 3 or 4 adverse events. The overall response rate was 41%, overall survival 18 months (95% confidence intervals 5-30 months) and progression free survival 7 months (95% CI 1-13), although there was a significant difference according to underlying hematological cancer subtype. Response rate was strongly associated with survival time. On multivariate analysis, disease subtype and LDH at time of trial entry showed an association with survival rate. These results demonstrate that the outcomes of patients with hematiological malignancies entering phase I trials is better than expected.