Anna Bosch-Vilaseca1, Irene García-Cadenas1, Elisa Roldán2, Silvana Novelli1, Pere Barba2, Albert Esquirol1, David Valcárcel2, Rodrigo Martino1, Jorge Sierra1. 1. Hematology Department, Hospital de la Santa Creu i Sant Pau, Biomedical Research Institute (IIB Sant-Pau) and José Carreras Leukemia Research Institute, Autonomous University of Barcelona (UAB), Barcelona, Spain. 2. Hematology Department, Hospital Vall d'Hebron, Experimental Hematology Unit, Vall d' Hebron Institute of Oncology (VHIO), Autonomous University of Barcelona (UAB), Barcelona, Spain.
Abstract
OBJECTIVE AND METHODS: Severe postengraftment thrombocytopenia is a common complication after allogeneic stem cell transplantation (alloSCT). A few studies have suggested that the use of thrombopoietin agonists (TPOa) may be useful in this setting. Our retrospective study is the largest series published to date; we retrospectively evaluated TPOa efficacy and safety in 20 adult alloSCT recipients who received TPOa as a compassionate use for clinically relevant thrombocytopenia. RESULTS: Twelve of 20 patients (60%) responded, with a 180-day cumulative incidence of successful platelet recovery to ≥30 and ≥50 × 109 /L of 57% (95% CI: 44%-71%) and 32% (95% CI: 18%-46%), respectively, which were reached at a median of 28 and 34 days from the start of therapy. Fifty percent of the responders were able to discontinue the TPOa without recurrence of severe thrombocytopenia and its associated hemorrhagic complications. No serious adverse events were reported. Possible variables associated with higher response to TPOa were as follows: age < 40 years, presence of megakaryocytes in the bone marrow aspirate, and/or prior response to other hematopoietic growth factors. CONCLUSION: This study adds further enthusiasm for continued research on the use of these agents for the treatment of persistent thrombocytopenia in alloSCT recipients.
OBJECTIVE AND METHODS: Severe postengraftment thrombocytopenia is a common complication after allogeneic stem cell transplantation (alloSCT). A few studies have suggested that the use of thrombopoietin agonists (TPOa) may be useful in this setting. Our retrospective study is the largest series published to date; we retrospectively evaluated TPOa efficacy and safety in 20 adult alloSCT recipients who received TPOa as a compassionate use for clinically relevant thrombocytopenia. RESULTS: Twelve of 20 patients (60%) responded, with a 180-day cumulative incidence of successful platelet recovery to ≥30 and ≥50 × 109 /L of 57% (95% CI: 44%-71%) and 32% (95% CI: 18%-46%), respectively, which were reached at a median of 28 and 34 days from the start of therapy. Fifty percent of the responders were able to discontinue the TPOa without recurrence of severe thrombocytopenia and its associated hemorrhagic complications. No serious adverse events were reported. Possible variables associated with higher response to TPOa were as follows: age < 40 years, presence of megakaryocytes in the bone marrow aspirate, and/or prior response to other hematopoietic growth factors. CONCLUSION: This study adds further enthusiasm for continued research on the use of these agents for the treatment of persistent thrombocytopenia in alloSCT recipients.
Authors: Marta Bortolotti; Loredana Pettine; Anna Zaninoni; Giorgio Alberto Croci; Wilma Barcellini; Bruno Fattizzo Journal: Pharmaceuticals (Basel) Date: 2022-03-30