Clinical trial NCT02063763: TPO-mimetics Before Splenectomy in Adult Primary Immune Thrombocytopenia Patients.
Official title: Use of TPO-mimetics to Prepare for Splenectomy in Adult Patients With Primary Immune Thrombocytopenia. Brooklyn Observational Retrospective Study.
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Adult Patients
Immune Primary Thrombocytopenia
Splenectomy
TPO-mimetics
Keywords: Adult patients
Immune primary thrombocytopenia
Splenectomy
TPO-mimetics
Eltrombopag
Romiplostim
Lead sponsor: Gruppo Italiano Malattie EMatologiche dell'Adulto
Sponsor class: OTHER
First posted: 2014-02-14
Enrollment: 31
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: CASE_ONLY
Time perspective: RETROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: ITP adult patients having undergone splenectomy after a bridge therapy with eltrombopag or romiplostim.

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Number of patients responding to TPO-mimetics
timeFrame: Six months.
Secondary outcomes:
measure: Number of patients on TPO-mimetics eltrombopag and romiplostim as bridge therapy before splenectomy.
timeFrame: Six months.
measure: Frequency and ways of administration.
timeFrame: Six months
measure: Number of hemorrhagic events.
timeFrame: At thirty days from splenectomy.
measure: Frequency of toxicity.
timeFrame: Six months
Other outcomes:
Locations:
city: Aversa
country: Italy
facility: U.O.C di Ematologia P.O. "S.Giuseppe Moscati"
city: Barletta
country: Italy
facility: UOC Ematologia Ospedale " Mons. Dimiccoli"
city: Firenze
country: Italy
facility: Policlinico di Careggi
city: Lecce
country: Italy
facility: ASL Le/1 P.O. Vito Fazzi - U.O. di Ematologia ed UTIE
city: Lido di Camaiore
country: Italy
facility: Unità Operativa Complessa) - Medicina Generale - Sezione di Ematologia - Ospedale Versilia USL 12 Toscana
city: Milano
country: Italy
facility: Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico UOC Oncoematologia- Padiglione Marcora 2° piano
city: Milano
country: Italy
facility: Ospedale Niguarda " Ca Granda" - SC Ematologia Blocco SUD, Ponti Est, Scala E, 4° piano
city: Milano
country: Italy
facility: Unità Trapianto di Midollo Ist. Nazionale Tumori
city: Monza
country: Italy
facility: Azienda Ospedaliera "S.Gerardo"
city: Novara
country: Italy
facility: S.C.D.U. Ematologia - DIMECS e Dipartimento Oncologico - Università del Piemonte Orientale Amedeo Avogadro
city: Roma
country: Italy
facility: Università degli Studi "Sapienza" - Dip Biotecnologie Cellulari ed Ematologia - Divisione di Ematologia
city: Terni
country: Italy
facility: A.O. Santa Maria - Terni S.C Oncoematologia
city: Udine
country: Italy
facility: Clinica Ematologica - Policlinico Universitario
city: Verona
country: Italy
facility: Università degli Studi di Verona - A. O. - Istituti Ospitalieri di Verona- Div. di Ematologia - Policlinico G.B. Rossi
Study officials:
affiliation: Clinica Ematologica, DISM, Azienda Ospedaliera Universitaria S. M. Misericordia
name: Francesco Zaja, Pr.
role: STUDY_CHAIR
References: