ITP0614: TPO-mimetics Before Splenectomy in Adult Primary Immune Thrombocytopenia Patients.

Sponsor

Gruppo Italiano Malattie EMatologiche dell'Adulto (Other)

Overall Status

Completed

CT.gov ID

NCT02063763

Collaborator

(none)

Enrollment

Locations

9.5

Actual Duration (Months)

2.2

Patients Per Site

0.2

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Adult Patients Immune Primary Thrombocytopenia Splenectomy TPO-mimetics

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 Design

Study Type:

Observational

Actual Enrollment :

31 participants

Observational Model:

Case-Only

Time Perspective:

Retrospective

Official Title:

Use of TPO-mimetics to Prepare for Splenectomy in Adult Patients With Primary Immune Thrombocytopenia. Brooklyn Observational Retrospective Study.

Actual Study Start Date :

May 14, 2014

Actual Primary Completion Date :

Feb 26, 2015

Actual Study Completion Date :

Feb 26, 2015

Outcome Measures

Primary Outcome Measures

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

Secondary Outcome Measures

Number of patients on TPO-mimetics eltrombopag and romiplostim as bridge therapy before splenectomy. [Six months.]
Frequency and ways of administration. [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

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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.

Contacts and Locations

Locations

	Site	City	Country
1	U.O.C di Ematologia P.O. "S.Giuseppe Moscati"	Aversa	Italy
2	UOC Ematologia Ospedale " Mons. Dimiccoli"	Barletta	Italy
3	Policlinico di Careggi	Firenze	Italy
4	ASL Le/1 P.O. Vito Fazzi - U.O. di Ematologia ed UTIE	Lecce	Italy
5	Unità Operativa Complessa) - Medicina Generale - Sezione di Ematologia - Ospedale Versilia USL 12 Toscana	Lido di Camaiore	Italy
6	Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico UOC Oncoematologia- Padiglione Marcora 2° piano	Milano	Italy
7	Ospedale Niguarda " Ca Granda" - SC Ematologia Blocco SUD, Ponti Est, Scala E, 4° piano	Milano	Italy
8	Unità Trapianto di Midollo Ist. Nazionale Tumori	Milano	Italy
9	Azienda Ospedaliera "S.Gerardo"	Monza	Italy
10	S.C.D.U. Ematologia - DIMECS e Dipartimento Oncologico - Università del Piemonte Orientale Amedeo Avogadro	Novara	Italy
11	Università degli Studi "Sapienza" - Dip Biotecnologie Cellulari ed Ematologia - Divisione di Ematologia	Roma	Italy
12	A.O. Santa Maria - Terni S.C Oncoematologia	Terni	Italy
13	Clinica Ematologica - Policlinico Universitario	Udine	Italy
14	Università degli Studi di Verona - A. O. - Istituti Ospitalieri di Verona- Div. di Ematologia - Policlinico G.B. Rossi	Verona	Italy