Thrombopoietin Receptor Agonists for Severe Thrombocytopenia after Allogeneic Stem Cell Transplantation: Experience of the Spanish Group of Hematopoietic Stem Cell Transplant |
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| Authors: | Leyre Bento José María Bastida Irene García-Cadenas Estefania García-Torres Daniel Rivera Anna Bosch-Vilaseca Carlos De Miguel María Esther Martínez-Muñoz Francesc Fernández-Avilés Elisa Roldán Anabelle Chinea Lucrecia Yáñez Teresa Zudaire Carlos Pinho Vaz Ildefonso Espigado Javier López David Valcárcel Rafael Duarte Antonia Sampol |
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| Affiliation: | 1. Hematology Department, Hospital Universitario Son Espases, Instituto de Investigación Sanitaria Islas Baleares, Palma de Mallorca, Spain;2. Hematology Department, Hospital Universitario de Salamanca, Salamanca, Spain;3. Hematology Department, Hospital Sant Creu i Sant Pau, Barcelona, Spain;4. Hematology Department, Hospital Universitario Reina Sofía, Córdoba, Spain;5. Hematology Department, Hospital Universitario Puerta de Hierro, Madrid, Spain;6. Hematology Department, Hospital Clínic de Barcelona, Barcelona, Spain;7. Hematology Department, Hospital Vall D''hebron, Barcelona, Spain;8. Hematology Department, Hospital Ramón y Cajal, Madrid, Spain;9. Hematology Department, Hospital Universitario Marqués de Valdecilla, Santander, Spain;10. Hematology Department, Complejo Hospitalario de Navarra, Pamplona, Spain;11. Hematology Department, Instituto Português de Oncologia, Oporto, Spain;12. Hematology Department, Hospital Virgen del Rocío, Sevilla, Spain;13. Hospital Clínico de Valencia, Valencia, Spain |
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| Abstract: | Persistent thrombocytopenia is a common complication after allogeneic hematopoietic stem cell transplantation (allo-SCT). Romiplostim and eltrombopag are the currently available thrombopoietin receptor agonists (TPO-RAs), and some studies with very small numbers of cases have reported their potential efficacy in the allo-SCT setting. The present retrospective study evaluated the safety and efficacy of TPO-RAs in 86 patients with persistent thrombocytopenia after allo-HSCT. Sixteen patients (19%) had isolated thrombocytopenia (PT), and 71 (82%) had secondary failure of platelet recovery (SFPR). TPO-RA therapy was started at a median of 127 days (range, 27 to 1177 days) after allo-SCT. The median initial and maximum administered doses were 50 mg/day (range, 25 to 150 mg/day) and 75 mg/day (range, 25 to 150 mg/day), respectively, for eltrombopag and 1 µg/kg (range, 1 to 7 µg/kg) and 5 µg/kg (range, 1 to 10 µg/kg), respectively, for romiplostin. The median platelet count before initiation of TPO-RA therapy was 14,000/µL (range, 1000 to 57,000/µL). Platelet recovery to ≥50,000/µL without transfusion support was achieved in 72% of patients at a median time of 66 days (range, 2 to 247 days). Eighty-one percent of the patients had a decreased number of megakaryocytes before treatment, showing a slower response to therapy (P = .011). The median duration of treatment was 62 days (range, 7 to 700 days). Grade 3-4 adverse events (hepatic and asthenia) were observed in only 2% of the patients. At last follow-up, 81% of patients had discontinued TPO-RAs and maintained response, and 71% were alive. To our knowledge, this is the largest series analyzing the use of TPO-RAs after allo-SCT reported to date. Our results support the efficacy and safety in this new setting. Further prospective trials are needed to increase the level of evidence and to identify predictors of response. |
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| Keywords: | Correspondence and reprint requests: Leyre Bento, MD, Hematology Department, Son Espases University Hospital, Ctra Valldemossa, 79, Planta +1, Módulo I, 07010 Palma, Illes Balears, Spain. Thrombocytopenia Allogeneic stem cell transplantation Thrombopoietin receptor agonists |
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