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INVESTIGATION AND MANAGEMENT OF PRIMARY IMMUNE THROMBOCYTOPENIA (ITP)

Aim

Provide up-to-date evidence and expert opinion on the management of patients with ITP, in order to reflect the numerous developments over the last decade.1

Panel

22 experts from 10 countries, including 18 adult haematologists, 3 paediatric haematologists and 1 patient representative.1,2

Recommendations for second-line ITP therapy1

  • Whilst corticosteroids are the standard first-line treatment for ITP they should only be used for a limited time (6–8 weeks) and most adults relapse upon cessation
    • Overuse and reliance on steroids are considered to be the most consistent and prevalent errors in ITP management
  • The goal of subsequent (second- and later-line) therapy is to provide a sustained increase in platelet count (that is considered haemostatic) whilst minimising adverse events and retaining the possibility of remission*
  • National licensing and availability, country-specific guidelines, cost, patient preference and profile (bleeding history, comorbidities and compliance) all influence treatment selection

Table of data highlighting the level of evidence for second-line treatment of ITP

  • The high costs associated with new treatments are compensated for by their efficacy, lack of immunosuppression and opportunity for treatment-free remission
  • Splenectomy is now only recommended after failure of medical therapies and is dependent on patient age and comorbidities

TPO-RAs FOR SUBSEQUENT (SECOND- AND LATER-LINE) ITP THERAPY

  • TPO-RAs (eltrombopag, avatrombopag and romiplostim) provide excellent responses regardless of splenectomy status for adults with ITP1
    • Eltrombopag has been associated with a 85.8% response rate (259/302) of achieving platelet counts ≥50x109/L1,3 and response appears to be unaffected by splenectomy status, baseline platelet count, concomitant ITP treatment or number of previous ITP treatments1,3–6
  • Response to TPO-RAs can persist for up to 8 years3 and often allows other ITP therapies to be reduced or discontinued1
  • Most adverse events are mild and the risk of clinically meaningful bone marrow reticulin fibrosis appears to be very low with TPO-RA treatment1,7,8
    • TPO-RAs are associated with a 6% incidence of arterial/venous thromboembolism, however, risk of thromboembolism is elevated in patients not undergoing TPO-RA therapy and TPO-RAs do not increase this risk vs. placebo3,9,10
  • Switching and sequential TPO-RA treatment has demonstrated positive effects on response and tolerability for adults and children with ITP1
  • ITP treatment response improves health-related QoL; this effect is greater for those receiving TPO-RAs than other therapies1
  • TPO-RAs provide good response and bleeding reduction with limited evidence of significant side effects for the majority of children with persistent or chronic ITP1  
    • Eltrombopag and romiplostim provide similar responses for children with primary or secondary or chronic ITP11

* Remission defined as platelet count ≥30 x109/L in the absence of ITP-specific treatment.3 †Not licensed in the UK.12,13

Abbreviations: ITP, immune thrombocytopenia; QoL, quality of life; TPO-RA, thrombopoietin-receptor agonist.

References

  1. Provan D, et al. Blood Adv. 2019;3(22):3780–817.
  2. Provan D, et al. Blood. 2010;115(2):168–86.
  3. Wong RSM, et al. Blood. 2017;130(23):2527–36.
  4. González-López TJ, et al. Int J Hematol. 2017;106(4):508–16.
  5. Bussel JB, et al. Lancet. 2009;373(9664):641–8.
  6. González-López TJ, et al. Eur J Haematol. 2016;97(3):297–302.
  7. Amgen Ltd. Nplate 125mcg powder for solution for injection vial. Summary of Product Characteristics. August 2018. Available at: https://www.medicines.org.uk/emc/product/9325/smpc (accessed December 2019).
  8. REVOLADE Summary of Product Characteristics.
  9. Cines DB, et al. Haematologica. 2017;102(8):1342–51.
  10. Sarpatwari A, et al. Haematologica. 2010;95(7):1167–75.
  11. Neunert C, et al. Pediatr Blood Cancer. 2016;63(8):1407–13.
  12. NICE. Proposed technology appraisal guidance GID-TA10348. June 2018. Available at: https://www.nice.org.uk/guidance/proposed/gid-ta10348/documents (accessed December 2019).
  13. NICE. Proposed technology appraisal guidance GID-TA10387. August 2019. Available at: https://www.nice.org.uk/guidance/proposed/gid-ta10387/documents (accessed December 2019).
HCP19-C003a March 2020.
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