The temporary use of thrombopoietin-receptor agonists may induce a prolonged remission in adult chronic immune thrombocytopenia. Results of a French observational study.
Thrombopoietin-receptor agonists (Tpo-RAs) are highly effective in immune thrombocytopenia (ITP). Recently, cases of durable remission after Tpo-RA discontinuation in adult ITP have been reported. We aimed to describe the subset of patients in whom transient Tpo-RA therapy may induce a durable response. We studied all adults with primary ITP treated with at least one Tpo-RA over a 5-year period (n = 54) and seen at one of three participating referral centres in France. Tpo-RAs were discontinued in 20 of 28 patients who achieved a complete response. We excluded six patients because a previous treatment at the start of Tpo-RA treatment may have interfered with the response. Overall, eight patients with chronic ITP showed a sustained response [median follow-up: 13·5 months (range 5-27 months)]. We could not identify a predictive factor of sustained response. In conclusion, a substantial proportion of ITP patients receiving Tpo-RAs can maintain a durable response after treatment discontinuation.
Service de Médecine Interne, Centre de référence des cytopénies auto-immunes de l'adulte, Hôpital Henri Mondor, Assistance Publique-Hôpitaux de Paris, Université Paris Est Créteil, Créteil, France; Etablissement Français du Sang Ile de France, Inserm U955, Créteil, Hôpital Henri Mondor, Paris, France.
Matthieu
M
Mahévas
Olivier
O
Fain
Mikael
M
Ebbo
Françoise
F
Roudot-Thoraval
Nicolas
N
Limal
Mehdi
M
Khellaf
Nicolas
N
Schleinitz
Philippe
P
Bierling
Laeticia
L
Languille
Bertrand
B
Godeau
Marc
M
Michel
D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D001565:Benzoates / Q000627:therapeutic use*; D004359:Drug Therapy, Combination; D005260:Female; D005602:France; D006801:Humans; D006834:Hydrazines / Q000627:therapeutic use*; D007155:Immunologic Factors / Q000627:therapeutic use; D008297:Male; D008875:Middle Aged; D016553:Purpura, Thrombocytopenic, Idiopathic / Q000188:drug therapy* / Q000601:surgery; D011720:Pyrazoles / Q000627:therapeutic use*; D011961:Receptors, Fc / Q000627:therapeutic use*; D053628:Receptors, Thrombopoietin / Q000819:agonists*; D011993:Recombinant Fusion Proteins / Q000627:therapeutic use*; D013156:Splenectomy; D013926:Thrombopoietin / Q000627:therapeutic use*; D016896:Treatment Outcome; D055815:Young Adult
D001565:Benzoates; D006834:Hydrazines; D007155:Immunologic Factors; D011720:Pyrazoles; D011961:Receptors, Fc; D053628:Receptors, Thrombopoietin; D011993:Recombinant Fusion Proteins; D013926:Thrombopoietin; C488777:romiplostim; C520809:eltrombopag
durable response; immune thrombocytopenia; prolonged remission; thrombopoietin-receptor agonists