EPIDEMIOLOGY AND MANAGEMENT OF PRIMARY IMMUNE THROMBOCYTOPENIA: REAL WORLD DATA FROM THE UK ITP REGISTRY
Author(s): ,
Abbas Zaidi
Affiliations:
Haematology,Royal London Hospital, BartsHealth NHS Trust ,London,United Kingdom
,
Caitlin Gracie
Affiliations:
Department of Immunobiology ,Queen Mary University of London,London,United Kingdom
,
Umesh Doobaree
Affiliations:
Department of Immunobiology ,Queen Mary University of London,London,United Kingdom
,
Drew Provan
Affiliations:
Department of Immunobiology ,Queen Mary University of London,London,United Kingdom
Mcdonald Vickie
Affiliations:
Haematology,Royal London Hospital, BartsHealth NHS Trust ,London,United Kingdom
(Abstract release date: 05/17/18) EHA Library. Zaidi A. 06/15/18; 215104; PF663
Abbas Zaidi
Abbas Zaidi
Contributions
Abstract

Abstract: PF663

Type: Poster Presentation

Presentation during EHA23: On Friday, June 15, 2018 from 17:30 - 19:00

Location: Poster area

Background
Immune thrombocytopenia (ITP) is a rare bleeding diathesis with an autoimmune background. Treatment practices vary considerably, there is a lack of epidemiological data and few large-scale randomised clinical trials to guide management.

Aims
We reviewed the UK Adult ITP registry, consisting of 89 sites across the United Kingdom who recruit cases of primary ITP, to describe real world characteristics of the population and evaluate management of ITP.

Methods
All clinical data from 2010- Jan 2018 was analysed for the diagnostic workup, bleeding symptoms, lines of treatment and clinical outcomes.

Results
2920 patients (57%  female) were entered into the registry in the time period. Median age at diagnosis was 50y (IQ range 35-66). The median platelet count at diagnosis was 19 x109/L  (IQR 5 - 57).  66% had bleeding symptoms (18.3% - 1 episode; 14.8% - 2 episodes; 32.8% ≥ 3 episodes) whereas 34% (995 patient) never had any bleeding. The commonest bleeding sites were: subcutaneous/ soft tissue (58.8%), oral ( 13.3%), epistaxis (11.6%) and gynaecological (4.9%). Intracranial haemorrhage occurred in 0.65% (0.44% non-traumatic) and all occurred within 6 months of diagnosis.Median Hb at diagnosis was 14.6g/L (IQR 12.4-14.6) , WCC 7.2 x10^9/L (IQR 5.5-9.9), neutrophils 4.4 x10^9/L. Median PT was 11.7s (IQR 10.9- 14.5) and APTT was 28.5s (IQR 25-31.8). 1171 patient had Antinuclear antibodies (ANA) sent, of these 14.6% were positive. 270 had Direct Antiglobulin Test (DAT) testing of which 2.59% were positive. Lupus anticoagulant testing was positive in 13.8% (n=577). Insufficient data was recorded for bone marrow testing to draw conclusions. Twenty seven percent (787) never required therapy for ITP. 21.6% (631) received 1 line of therapy, 14.6% received 2 lines and  and 19.9%% were treated with ≥3 lines of therapy. The most frequent treatment was steroids (65% prednisolone, 4.11% methylprednisolone and 9.25% dexamethasone) followed by IVIG (31.47%), rituximab (17.25%), MMF (11.7%), romiplostim (9.6%) and eltrombopag (5.99%). Splenectomy was performed in 9.83%.Outcomes and follow-up (6 months, 1 year, 5 years) are shown in table 1. We compared partial response (PR, platelet count 30-100x109/L) and complete response (CR, platelet count > 100 x109/L) in those who required no therapy, 1 and  ≥ 2 lines of treatment over time.The proportion of patients in the total cohort with severe thrombocytopenia (platelet < 10 x109/L) at 1 year was 3.1% and at 5 years, 2.7%. PR and CR rates one year from diagnosis were  44.7% and 41.9% respectively with 83.6% having a Platelet count > 30 x109/L at 5 years. A greater percentage of patients were in PR/CR in those who were untreated vs those who required ≥2 lines of therapy (96% vs 79%). 1.37% of patients still had bleeding symptoms after 5 years.

Conclusion
These data describe characteristics and outcome for patients with primary ITP in the UK population. Although limited by being observational and retrospective, they demonstrate that approximately 1/3 patients with primary ITP do not need treatment and currently, steroids and IVIG are the most common treatments used. Approximately 20% of patients require additional treatment beyond this including  immunosuppressive therapy and thrombopoeitin agonists. Approximately 10% require splenectomy. Longer term follow up of upto 5 years from diagnosis shows more than 80% of patients will be at least PR  (platelet count >30 x109/L) and fewer than 2% of patients will have ongoing bleeding symptoms.

Session topic: 33.  Platelets disorders

Keyword(s): Idiopathic thombocytopenic purpura (ITP)

Abstract: PF663

Type: Poster Presentation

Presentation during EHA23: On Friday, June 15, 2018 from 17:30 - 19:00

Location: Poster area

Background
Immune thrombocytopenia (ITP) is a rare bleeding diathesis with an autoimmune background. Treatment practices vary considerably, there is a lack of epidemiological data and few large-scale randomised clinical trials to guide management.

Aims
We reviewed the UK Adult ITP registry, consisting of 89 sites across the United Kingdom who recruit cases of primary ITP, to describe real world characteristics of the population and evaluate management of ITP.

Methods
All clinical data from 2010- Jan 2018 was analysed for the diagnostic workup, bleeding symptoms, lines of treatment and clinical outcomes.

Results
2920 patients (57%  female) were entered into the registry in the time period. Median age at diagnosis was 50y (IQ range 35-66). The median platelet count at diagnosis was 19 x109/L  (IQR 5 - 57).  66% had bleeding symptoms (18.3% - 1 episode; 14.8% - 2 episodes; 32.8% ≥ 3 episodes) whereas 34% (995 patient) never had any bleeding. The commonest bleeding sites were: subcutaneous/ soft tissue (58.8%), oral ( 13.3%), epistaxis (11.6%) and gynaecological (4.9%). Intracranial haemorrhage occurred in 0.65% (0.44% non-traumatic) and all occurred within 6 months of diagnosis.Median Hb at diagnosis was 14.6g/L (IQR 12.4-14.6) , WCC 7.2 x10^9/L (IQR 5.5-9.9), neutrophils 4.4 x10^9/L. Median PT was 11.7s (IQR 10.9- 14.5) and APTT was 28.5s (IQR 25-31.8). 1171 patient had Antinuclear antibodies (ANA) sent, of these 14.6% were positive. 270 had Direct Antiglobulin Test (DAT) testing of which 2.59% were positive. Lupus anticoagulant testing was positive in 13.8% (n=577). Insufficient data was recorded for bone marrow testing to draw conclusions. Twenty seven percent (787) never required therapy for ITP. 21.6% (631) received 1 line of therapy, 14.6% received 2 lines and  and 19.9%% were treated with ≥3 lines of therapy. The most frequent treatment was steroids (65% prednisolone, 4.11% methylprednisolone and 9.25% dexamethasone) followed by IVIG (31.47%), rituximab (17.25%), MMF (11.7%), romiplostim (9.6%) and eltrombopag (5.99%). Splenectomy was performed in 9.83%.Outcomes and follow-up (6 months, 1 year, 5 years) are shown in table 1. We compared partial response (PR, platelet count 30-100x109/L) and complete response (CR, platelet count > 100 x109/L) in those who required no therapy, 1 and  ≥ 2 lines of treatment over time.The proportion of patients in the total cohort with severe thrombocytopenia (platelet < 10 x109/L) at 1 year was 3.1% and at 5 years, 2.7%. PR and CR rates one year from diagnosis were  44.7% and 41.9% respectively with 83.6% having a Platelet count > 30 x109/L at 5 years. A greater percentage of patients were in PR/CR in those who were untreated vs those who required ≥2 lines of therapy (96% vs 79%). 1.37% of patients still had bleeding symptoms after 5 years.

Conclusion
These data describe characteristics and outcome for patients with primary ITP in the UK population. Although limited by being observational and retrospective, they demonstrate that approximately 1/3 patients with primary ITP do not need treatment and currently, steroids and IVIG are the most common treatments used. Approximately 20% of patients require additional treatment beyond this including  immunosuppressive therapy and thrombopoeitin agonists. Approximately 10% require splenectomy. Longer term follow up of upto 5 years from diagnosis shows more than 80% of patients will be at least PR  (platelet count >30 x109/L) and fewer than 2% of patients will have ongoing bleeding symptoms.

Session topic: 33.  Platelets disorders

Keyword(s): Idiopathic thombocytopenic purpura (ITP)

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