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Development and validation of a model to predict platelet response to romiplostim in patients with lower‐risk myelodysplastic syndromes
Author(s) -
Sekeres Mikkael A.,
Giagounidis Aristoteles,
Kantarjian Hagop,
Mufti Ghulam J.,
Fenaux Pierre,
Jia Catherine,
Yang Allen S.,
Platzbecker Uwe
Publication year - 2014
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.13037
Subject(s) - romiplostim , medicine , thrombopoietin , platelet , myelodysplastic syndromes , eltrombopag , international prognostic scoring system , cohort , platelet transfusion , immunology , bone marrow , haematopoiesis , genetics , stem cell , immune thrombocytopenia , biology
Summary Low endogenous erythropoietin levels and limited red blood cell transfusion history can predict response to erythropoiesis‐stimulating agents in anaemic patients with myelodysplastic syndromes ( MDS ). The relationship between endogenous thrombopoietin ( THPO ) levels and platelet response to romiplostim is unknown. Variables including baseline endogenous THPO levels, transfusion needs, and platelet response were analysed in a randomized trial of 250 thrombocytopenic, lower‐risk MDS patients (International Prognostic Scoring System low/intermediate‐1). A predictive scoring system was developed based on log–likelihood ratios and logistic coefficients. Patients with HI –P (haematological improvement – platelets) responses had lower mean baseline THPO levels ( P = 0·0497) and were more likely to have <6 platelet units transfused in the past year ( P = 0·0027), as did patients with platelet responses ≥50% of weeks on romiplostim ( P = 0·001 and P = 0·0037, respectively). A model for predicting response to romiplostim was developed and validated in a separate MDS cohort ( N = 72). Patients in low‐, intermediate‐, and high‐response groups had response rates of 17·4%, 29·6%, and 50·7%, respectively, for HI ‐P, and 17·4%, 33·8%, and 65·2%, respectively, for ≥50% response. For thrombocytopenic patients with lower‐risk MDS , lower baseline THPO levels (<500 pg/ml) and limited platelet transfusion history predicted a greater likelihood of a subsequent platelet response to romiplostim.