Successful discontinuation of eltrombopag after complete remission in patients with primary immune thrombocytopenia.
Eltrombopag is effective and safe in immune thrombocytopenia (ITP). Some patients may sustain their platelet response when treatment is withdrawn but the frequency of this phenomenon is unknown. We retrospectively evaluated 260 adult primary ITP patients (165 women and 95 men; median age, 62 years) treated with eltrombopag after a median time from diagnosis of 24 months. Among the 201 patients who achieved a complete remission (platelet count >100 × 10(9) /l), eltrombopag was discontinued in 80 patients. Reasons for eltrombopag discontinuation were: persistent response despite a reduction in dose over time (n = 33), platelet count >400 × 10(9) /l (n = 29), patient's request (n = 5), elevated aspartate aminotransferase (n = 3), diarrhea (n = 3), thrombosis (n = 3), and other reasons (n = 4). Of the 49 evaluable patients, 26 patients showed sustained response after discontinuing eltrombopag without additional ITP therapy, with a median follow-up of 9 (range, 6-25) months. These patients were characterized by a median time since ITP diagnosis of 46.5 months, with 4/26 having ITP < 1 year. Eleven patients were male and their median age was 59 years. They received a median of 4 previous treatment lines and 42% were splenectomized. No predictive factors of sustained response after eltrombopag withdrawal were identified. Platelet response following eltrombopag cessation may be sustained in an important percentage of adult primary ITP patients who achieved CR with eltrombopag. However, reliable markers for predicting which patients will have this response are needed.
Department of Hematology, Hospital Universitario de Burgos, Burgos, Spain.
Tomás José
TJ
González-López
Cristina
C
Pascual
María Teresa
MT
Álvarez-Román
Fernando
F
Fernández-Fuertes
Blanca
B
Sánchez-González
Isabel
I
Caparrós
Isidro
I
Jarque
María Eva
ME
Mingot-Castellano
José Angel
JA
Hernández-Rivas
Mónica
M
Martín-Salces
Laura
L
Solán
Paola
P
Beneit
Reyes
R
Jiménez
Silvia
S
Bernat
Marcio M
MM
Andrade
Montserrat
M
Cortés
Maria José
MJ
Cortti
Susana
S
Pérez-Crespo
Marta
M
Gómez-Núñez
Pavel E
PE
Olivera
Gloria
G
Pérez-Rus
Violeta
V
Martínez-Robles
Rafael
R
Alonso
Angeles
A
Fernández-Rodríguez
María Carmen
MC
Arratibel
María
M
Perera
Carmen
C
Fernández-Miñano
Miguel Angel
MA
Fuertes-Palacio
Juan Andrés
JA
Vázquez-Paganini
Isabel
I
Gutierrez-Jomarrón
Inés
I
Valcarce
Erik
E
de Cabo
Adriana
A
Sainz
Rosa
R
Fisac
Carlos
C
Aguilar
María
M
Paz Martínez-Badas
María Jesús
MJ
Peñarrubia
María
M
Calbacho
Carmen
C
de Cos
Manuel
M
González-Silva
Erika
E
Coria
Arancha
A
Alonso
Alberto
A
Casaus
Armando
A
Luaña
Pilar
P
Galán
Cristina
C
Fernández-Canal
Javier
J
Garcia-Frade
José Ramón
JR
González-Porras
D000328:Adult; D000368:Aged; D001565:Benzoates / Q000008:administration & dosage*; D001792:Blood Platelets / Q000187:drug effects / Q000473:pathology; D002908:Chronic Disease; D004334:Drug Administration Schedule; D016903:Drug Monitoring; D004920:Erythropoiesis / Q000187:drug effects*; D005260:Female; D005500:Follow-Up Studies; D006397:Hematinics / Q000008:administration & dosage*; D006801:Humans; D006834:Hydrazines / Q000008:administration & dosage*; D008297:Male; D008875:Middle Aged; D010976:Platelet Count; D016553:Purpura, Thrombocytopenic, Idiopathic / Q000188:drug therapy* / Q000473:pathology / Q000601:surgery; D011720:Pyrazoles / Q000008:administration & dosage*; D053628:Receptors, Thrombopoietin / Q000819:agonists / Q000235:genetics / Q000378:metabolism; D012008:Recurrence; D012074:Remission Induction; D012189:Retrospective Studies; D013156:Splenectomy; D016896:Treatment Outcome
D001565:Benzoates; D006397:Hematinics; D006834:Hydrazines; D011720:Pyrazoles; D053628:Receptors, Thrombopoietin; C520809:eltrombopag