Clinical Trial Details
— Status: Recruiting
Administrative data
| NCT number |
NCT03465020 |
| Other study ID # |
ITP0918 |
| Secondary ID |
|
| Status |
Recruiting |
| Phase |
|
| First received |
|
| Last updated |
|
| Start date |
October 15, 2018 |
| Est. completion date |
July 2023 |
Study information
| Verified date |
July 2022 |
| Source |
Gruppo Italiano Malattie EMatologiche dell'Adulto |
| Contact |
Paola Fazi |
| Phone |
+39 06.70390521 |
| Email |
p.fazi[@]gimema.it |
| Is FDA regulated |
No |
| Health authority |
|
| Study type |
Observational [Patient Registry]
|
Clinical Trial Summary
The objective of the study is to produce a dynamic picture of the adult ITP natural history
and management in Italy by collecting standardized information retrospective and prospective
data of patients in active management through a nation-wide registry.
Description:
Primary immune thrombocytopenia (ITP) is an acquired autoimmune disorder affecting both
children and adults, characterized by a platelet count below 100 x 109/L in the absence of
any underlying disease explaining the thrombocytopenia. Clinical manifestations may be absent
or include cutaneous mucosal or organ bleeding, resulting in an overall mortality rate of
less than 1-2%.
Previously, 'acute ITP' was used to describe a self-limited form of the disease and 'chronic
ITP' to describe the disease if it lasted for more than 6 months. In 2009, a new terminology
for ITP was agreed upon based on the duration of the disease. The new terms for ITP are:
'newly diagnosed ITP (from diagnosis to 3 months), 'persistent ITP' (3-12 months) and
'chronic ITP' (lasting for more than 12 months) [2].
Chronic primary ITP in adults is slightly more common in women of childbearing age but the
sex incidence is similar in patients over 60. The incidence of ITP in adults is around 4 per
100.000 people per year, with a UK prevalence of up to 24 per 100.000 people [4-6]. No more
than 2 per 100.000 will require ITP treatment.
Bleeding is highly variable and there is great heterogeneity. Bleeding is most commonly
mucocutaneous, involving the skin and subcutaneous tissue (petechiae, ecchymoses,
subcutaneous hematomas), the external mucosae (epistaxis, gum bleeding, blood blisters in the
mouth), muscles (muscle hematomata) or the various organs and internal mucosae (hematuria,
gastrointestinal bleeding, menorrhagia, intracerebral bleeding). Major bleeding is not common
if the platelet count is above 30x109/L. Intracranial hemorrhage is rare and is most often
seen in older patients who have additional comorbidities and in patients who fail to respond
to therapy. The yearly risk of fatal hemorrhage is around 1.6-3.9% in unresponsive patients.
This risk varies with age, from 0.4% per year in patients below 40 years old to 13% per year
in those over 60. The natural history of ITP with currently available treatments is not well
known. Available data are mostly based on the era when rituximab and thrombopoietin-receptor
agonists (TPO-RA) were not in use.
There is a lack reliable data on how recent treatments have modified the course of the
disease. Bleeding frequency and severity, complications like infections, thrombosis,
ITP-related symptoms like fatigue, etc. are also largely unknown as are women's related
issues. Similarly, the cost of treatment for a patient with ITP is not available. Safety and
efficacy of specific recent treatments have been measured mostly in industry-sponsored
studies. Furthermore, most studies did not conform to the International Working Group (IWG)
on ITP recommendations concerning terminology, definitions, outcome criteria and assessment
of bleeding.
To the investigator's knowledge, an ITP Registry is ongoing in UK and in Australia and
registries are being planned in other European countries with a prospective of creating an
international network. The only established international registry, the ITP PARC study, is
operative since 2004 but covers different aspects of the disease. The few Italian registries
are of regional dimension and are not aligned to collect standardized information in a
uniform way.
