title: The use of thrombopoietin-receptor agonists (TPO-RAs) in immune thrombocytopenia (ITP): a "real life" retrospective multicenter experience of the Rete Ematologica Pugliese (REP).
abstract: Immune thrombocytopenia (ITP) is a disease which sees one-third of patients failing first and subsequent therapeutic approaches, including splenectomy. Thrombopoietin-receptor agonists (TPO-RAs) are recommended for adults who relapse after splenectomy or who have contraindications for splenectomy. In this multicenter study, a total of 124 patients were retrospectively evaluated: 55 (44.3 %) were treated by romiplostim and 69 (55.6 %) by eltrombopag. Mean age, number of young patients (<60 years), time from primary diagnosis of ITP to TPO-RA treatment, and previous lines of therapy were similar in both groups. The overall response rate was 80 % (44/55) for romiplostim and 94.2 % (65/69) for eltrombopag; the duration of response and the time to response were similar (p = NS). The response rate to both drugs in non-splenectomized patients was higher than that of splenectomized patients (p < 0.05). The mean duration of response was 30 months for romiplostim and 15 months for eltrombopag, due to later commercialization of eltrombopag. Failure was the most frequent cause of discontinuation. Thrombotic events were the most consistent adverse events and were recorded in 2 and 3 % of patients treated by romiplostim and eltrombopag, respectively. In conclusion, romiplostim and eltrombopag are effective in the majority of patients with chronic ITP who failed several lines of therapy; whether TPO-RAs could substitute splenectomy is under discussion and studies are warranted.
authors.affiliation: Haematology Unit and BMT, "San Giuseppe Moscati" Hospital, Taranto, Italy.
authors.forename: Patrizio
authors.initials: P
authors.lastname: Mazza
authors.affiliation: Haematology Unit and BMT, "San Giuseppe Moscati" Hospital, Taranto, Italy. carlaminoia@libero.it.
authors.forename: Carla
authors.initials: C
authors.lastname: Minoia
authors.affiliation: Haematology Unit, "Antonio Perrino" Hospital, Brindisi, Italy.
authors.forename: Angela
authors.initials: A
authors.lastname: Melpignano
authors.affiliation: Haematology Unit, "F. Miulli" Hospital, Acquaviva delle Fonti, Bari, Italy.
authors.forename: Giuseppe
authors.initials: G
authors.lastname: Polimeno
authors.affiliation: Division of Hematology, Casa Sollievo della Sofferenza Hospital, IRCCS, San Giovanni Rotondo, Italy.
authors.forename: Nicola
authors.initials: N
authors.lastname: Cascavilla
authors.affiliation: Haematology Unit and BMT, "V. Fazzi" Hospital, Lecce, Italy.
authors.forename: Nicola
authors.initials: N
authors.lastname: Di Renzo
authors.affiliation: Unit of Hematology with BMT, Department of Emergency and Organ Transplantation, University of Bari, Bari, Italy.
authors.forename: Giorgina
authors.initials: G
authors.lastname: Specchia
mesh_terms: D000368:Aged; D001565:Benzoates / Q000494:pharmacology / Q000627:therapeutic use*; D015331:Cohort Studies; D005260:Female; D006761:Hospitals / Q000639:trends; D006801:Humans; D006834:Hydrazines / Q000494:pharmacology / Q000627:therapeutic use*; D007558:Italy / Q000453:epidemiology; D008297:Male; D008875:Middle Aged; D016553:Purpura, Thrombocytopenic, Idiopathic / Q000175:diagnosis / Q000188:drug therapy* / Q000453:epidemiology*; D011720:Pyrazoles / Q000494:pharmacology / Q000627:therapeutic use*; D011961:Receptors, Fc / Q000627:therapeutic use*; D053628:Receptors, Thrombopoietin / Q000819:agonists*; D011993:Recombinant Fusion Proteins / Q000494:pharmacology / Q000627:therapeutic use*; D012189:Retrospective Studies; D013926:Thrombopoietin / Q000494:pharmacology / Q000627:therapeutic use*
chemical_list: D001565:Benzoates; D006834:Hydrazines; D011720:Pyrazoles; D011961:Receptors, Fc; D053628:Receptors, Thrombopoietin; D011993:Recombinant Fusion Proteins; C082218:MPL protein, human; D013926:Thrombopoietin; C488777:romiplostim; C520809:eltrombopag
keywords: Immune thrombocytopenia (ITP); Splenectomy; Thrombopoietin receptor agonists