TPO-mimetics Before Splenectomy in Adult Primary Immune Thrombocytopenia Patients.

Completed

• Conditions: TPO-mimetics; Adult Patients; Immune Primary Thrombocytopenia; Splenectomy

• Registration Number: NCT02063763
• Lead Sponsor: Gruppo Italiano Malattie EMatologiche dell'Adulto

Brief Summary

This study aims at analyzing the therapeutic activity of TPO-mimetics Eltrombopag and Romiplostim as bridge therapy for splenectomy in adult patients with primary immune thrombocytopenia.

Detailed Description

Adult immune primary thrombocytopenia is an autoimmune malignancy characterized by platelet destruction and inadequate platelet production. Its incidence is of three cases per 100,000 people a year, with a prevalence in women in young and older adults.

Splenectomy is, still today, the therapeutic approach that offers a bigger guarantee of a long term response (around 60+70%). Nevertheless, splenectomy may be accompanied by peri-operative complications in almost 10% of patients, which in rare cases may be fatal. These are normally hemorrhagic complications due to low platelet count. Thus, a previous therapy to increase platelets is advisable before any splenectomy.

In patients who are refractory to corticosteroids and immunoglobulins or when its use is not indicated, splenectomy has a potential risk of more complications. Since the last few years, we count with TPO-mimetic drugs, specific for c-MPL receptor, able to stimulate platelet production, such as romiplostim and eltrombopag.

Nowadays, TPO-mimetics are allowed in Italy when patients refuse splenectomy or when splenectomy is not contraindicated, but being ITP with a low platelet count (\< 20-50.000/mmc) a potential contraindication to splenectomy due to hemorrhagic events, these drugs should be considered. Nevertheless, there are no data on therapeutic risks and safety of these agents when used for this indication. The aim of the present study is to verify, on an Italian national scale, the frequency of use, its impact and the safety profile.

Study Timeline

• Study First Submitted: 2014-02-13
• Study First Submitted QC: 2014-02-13
• Study First Posted: 2014-02-14
• Study Start: 2014-05-14
• Primary Completion: 2015-02-26
• Study Completion: 2015-02-26
• Status Verified: 2020-10
• Last Update Submitted: 2020-10-12
• Last Update Posted: 2020-10-14

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 31

Inclusion Criteria

• Persistent or chronic ITP in symtomatic phase.
• 18 years of age or older.
• Indication for splenectomy due to refractory response to a previous therapy.
• Have used eltrombopag or romiplostim to increase platelet count before splenectomy.
• Have undergone splenectomy.

Exclusion Criteria

• No informed consent.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Number of patients responding to TPO-mimetics	Six months.	According to platelet count and presence or absence of hemorrhagic events.

Secondary Outcome Measures

Name	Time	Method
Frequency and ways of administration.	Six months
Number of patients on TPO-mimetics eltrombopag and romiplostim as bridge therapy before splenectomy.	Six months.
Number of hemorrhagic events.	At thirty days from splenectomy.	Number and seriousness of hemorrhagic, thrombotic and infectious events at 30 days from splenectomy.
Frequency of toxicity.	Six months	NCI CTCAE v. 4.0

Trial Locations

Locations (14)

U.O.C di Ematologia P.O. "S.Giuseppe Moscati"; 🇮🇹 Aversa, Italy

ASL Le/1 P.O. Vito Fazzi - U.O. di Ematologia ed UTIE; 🇮🇹 Lecce, Italy

UOC Ematologia Ospedale " Mons. Dimiccoli"; 🇮🇹 Barletta, Italy

Policlinico di Careggi; 🇮🇹 Firenze, Italy

Unità Operativa Complessa) - Medicina Generale - Sezione di Ematologia - Ospedale Versilia USL 12 Toscana; 🇮🇹 Lido di Camaiore, Italy

Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico UOC Oncoematologia- Padiglione Marcora 2° piano; 🇮🇹 Milano, Italy

Ospedale Niguarda " Ca Granda" - SC Ematologia Blocco SUD, Ponti Est, Scala E, 4° piano; 🇮🇹 Milano, Italy

Unità Trapianto di Midollo Ist. Nazionale Tumori; 🇮🇹 Milano, Italy

Azienda Ospedaliera "S.Gerardo"; 🇮🇹 Monza, Italy

S.C.D.U. Ematologia - DIMECS e Dipartimento Oncologico - Università del Piemonte Orientale Amedeo Avogadro; 🇮🇹 Novara, Italy

A.O. Santa Maria - Terni S.C Oncoematologia; 🇮🇹 Terni, Italy

Università degli Studi "Sapienza" - Dip Biotecnologie Cellulari ed Ematologia - Divisione di Ematologia; 🇮🇹 Roma, Italy

Clinica Ematologica - Policlinico Universitario; 🇮🇹 Udine, Italy

Università degli Studi di Verona - A. O. - Istituti Ospitalieri di Verona- Div. di Ematologia - Policlinico G.B. Rossi; 🇮🇹 Verona, Italy