Safety and efficacy of eltrombopag for treatment of chronic immune thrombocytopenia: results of the long-term, open-label EXTEND study
Mansoor N Saleh, James B Bussel, Gregory Cheng, Oliver Meyer, Christine K Bailey, Michael Arning, Andres Brainsky, EXTEND Study Group, B Chong, M Pidcock, I Pabinger-Fasching, H Olney, D Arnold, S Assouline, Z Shen, R Yang, A Sun, J Mayer, V Pohlreichova, A Hlusi, C Marcher, T Nousiainen, B Godeau, S Cheze, J Viallard, A Ganser, A Matzdorff, A Salama, D Schroeder, D Schroeder, M von Depka Prondzinski, E Hiller, M Rummel, A Anagnostopoulos, C Poziopoulos, R Wong, F Fabris, F Ciceri, F Angelini, N Vianelli, B Kim, V Novotný, B J Biemond, M R Schipperus, S Wittebol, H Blacklock, D Simpson, P Ganly, R Doocey, T Shamsi, Z Aziz, S Murillo, V Ulloa, J Kloczko, A Dmoszynska, T Robak, K Kuliczkowski, W Homenda, M Calbecka, R Antonio Stoia, A Vladareanu, L Kovaleva, O Rukavitsyn, N Domnikova, K Abdulkadyrov, S Palasthy, J A Reimana, J Lazur, A Escudero Soto, J Besalduch, J Páramo Fernández, S Gerosa, J Ribera Santasusana, S Perez Crespo, A Alonso, C Pascual Izquierdo, H Wadenvik, R Lerner, W Tsay, P Rojnuckarin, C Sirijerachai, B Meddeb, F M'Sadek, A Khelif, S Davies, T Nokes, H Grech, S Al-Ismail, M Scully, V Martlew, Z Masliak, P Kaplan, T Cosgriff, R Giudice, J Hon, P Kuriakose, H Liebman, P Flynn, D Smith, P Fogarty, D Quick, M de Oliveira, M Taylor, T Gernsheimer, V Pullarkat, M Rarick, C Scroggin Jr, J George, Y Soong Ahn, D Kuter, M Tarantino, K McCrae, L Phan, Mansoor N Saleh, James B Bussel, Gregory Cheng, Oliver Meyer, Christine K Bailey, Michael Arning, Andres Brainsky, EXTEND Study Group, B Chong, M Pidcock, I Pabinger-Fasching, H Olney, D Arnold, S Assouline, Z Shen, R Yang, A Sun, J Mayer, V Pohlreichova, A Hlusi, C Marcher, T Nousiainen, B Godeau, S Cheze, J Viallard, A Ganser, A Matzdorff, A Salama, D Schroeder, D Schroeder, M von Depka Prondzinski, E Hiller, M Rummel, A Anagnostopoulos, C Poziopoulos, R Wong, F Fabris, F Ciceri, F Angelini, N Vianelli, B Kim, V Novotný, B J Biemond, M R Schipperus, S Wittebol, H Blacklock, D Simpson, P Ganly, R Doocey, T Shamsi, Z Aziz, S Murillo, V Ulloa, J Kloczko, A Dmoszynska, T Robak, K Kuliczkowski, W Homenda, M Calbecka, R Antonio Stoia, A Vladareanu, L Kovaleva, O Rukavitsyn, N Domnikova, K Abdulkadyrov, S Palasthy, J A Reimana, J Lazur, A Escudero Soto, J Besalduch, J Páramo Fernández, S Gerosa, J Ribera Santasusana, S Perez Crespo, A Alonso, C Pascual Izquierdo, H Wadenvik, R Lerner, W Tsay, P Rojnuckarin, C Sirijerachai, B Meddeb, F M'Sadek, A Khelif, S Davies, T Nokes, H Grech, S Al-Ismail, M Scully, V Martlew, Z Masliak, P Kaplan, T Cosgriff, R Giudice, J Hon, P Kuriakose, H Liebman, P Flynn, D Smith, P Fogarty, D Quick, M de Oliveira, M Taylor, T Gernsheimer, V Pullarkat, M Rarick, C Scroggin Jr, J George, Y Soong Ahn, D Kuter, M Tarantino, K McCrae, L Phan
Abstract
Patients with chronic immune thrombocytopenia may have bleeding resulting from low platelet counts. Eltrombopag increases and maintains hemostatic platelet counts; however, to date, outcome has been reported only for treatment lasting ≤ 6 months. This interim analysis of the ongoing open-label EXTEND (Eltrombopag eXTENded Dosing) study evaluates the safety and efficacy of eltrombopag in 299 patients treated up to 3 years. Splenectomized and nonsplenectomized patients achieved platelets ≥ 50 000/μL at least once (80% and 88%, respectively). Platelets ≥ 50 000/μL and 2 × baseline were maintained for a median of 73 of 104 and 109 of 156 cumulative study weeks, respectively. Bleeding symptoms (World Health Organization Grades 1-4) decreased from 56% of patients at baseline to 20% at 2 years and 11% at 3 years. One hundred (33%) patients were receiving concomitant treatments at study entry, 69 of whom attempted to reduce them; 65% (45 of 69) had a sustained reduction or permanently stopped ≥ 1 concomitant treatment. Thirty-eight patients (13%) experienced ≥ 1 adverse events leading to study withdrawal, including patients meeting protocol-defined withdrawal criteria (11 [4%] thromboembolic events, 5 [2%] exceeding liver enzyme thresholds). No new or increased incidence of safety issues was identified. Long-term treatment with eltrombopag was generally safe, well tolerated, and effective in maintaining platelet counts in the desired range.
Trial registration: ClinicalTrials.gov NCT00351468.
Source: PubMed