1.Have splenectomy rate and main outcomes of ITP changed after the introduction of new treatments? A monocentric study in the outpatient setting during 35 years.
Palandri F1, Polverelli N1, Sollazzo D1, Romano M1, Catani L1, Cavo M1, Vianelli N1. Am J Hematol. 2016 Jun;91(4):E267-72. doi: 10.1002/ajh.24310.
In the last years, rituximab (RTX) and agonists of the thrombopoietin receptor (TPO-R) eltrombopag and romiplostim have provided new treatment options in persistent and chronic immune thrombocytopenia (ITP). Here, we analyzed the changes in therapeutic choices over time and their impact on clinical outcomes in a cohort of 557 ITP outpatients followed at the "L. and A. Seràgnoli" Institute of Hematology, Bologna, Italy, from 1980 to 2015. Overall 397 patients (71%) required front-line corticosteroids, mainly prednisone. Over the decades, splenectomy was delayed from second to third-line, but was steadily used in around 15-25% of patients refractory or relapsing after first-line treatment. Consensually, RTX and TPO-R agonists emerged as second and third-line therapy of choice, respectively. Splenectomy was associated with the best response rates and the lower incidences of relapse, while the relapse rate after RTX was comparable to that observed with corticosteroids and other immunosuppressive agents.
2.Health-related quality of life in thrombocytopenic patients with chronic hepatitis C with or without cirrhosis in the ENABLE-1 and ENABLE-2 studies.
Grotzinger KM1, Younossi ZM2, Giannini EG3, Chen PJ4, Rendas-Baum R5, Theodore D6. Health Qual Life Outcomes. 2016 Mar 22;14(1):49. doi: 10.1186/s12955-016-0447-1.
BACKGROUND: Despite changes in the treatment paradigm towards non-interferon-based therapies, interferon-based treatments are still used in some geographical regions for treating patients with hepatitis C virus (HCV) infection. Use of eltrombopag with interferon-based treatment for patients with thrombocytopenia and HCV was assessed in two similarly designed phase 3 trials (Eltrombopag to Initiate and Maintain Interferon Antiviral Treatment to Benefit Subjects With Hepatitis C-Related Liver Disease [ENABLE-1 and ENABLE-2]). These trials also aimed to determine whether response to antiviral therapy (e.g., sustained virologic response [SVR]) is associated with changes in health-related quality of life (HRQoL). This pooled, post-hoc analysis aimed to (1) determine whether or not specific aspects of clinical response to treatment (i.e., achieving SVR) are associated with a significant change in HRQoL, and (2) to determine the magnitude and direction of the association between important changes in HRQoL, clinical response to interferon-based therapy (e.
3.Thrombopietin receptor agonists for preparing adult patients with immune thrombocytopenia to splenectomy. Results of a retrospective, observational GIMEMA study.
Zaja F1, Barcellini W2, Cantoni S3, Carpenedo M4, Caparrotti G5, Carrai V6, Di Renzo N7, Santoro C8, Di Nicola M9, Veneri D10, Simonetti F11, Liberati AM12, Ferla V2, Paoloni F13, Crea E13, Volpetti S1, Tuniz E1, Fanin R1. Am J Hematol. 2016 Feb 22. doi: 10.1002/ajh.24341. [Epub ahead of print]
In patients with immune thrombocytopenia (ITP) refractory to corticosteroids and intravenous immunoglobulins (IGIV), splenectomy may result at higher risk of peri-operative complications and, for this reason, potentially contraindicated. The thrombopoietin receptor agonists (TPO-RAs) romiplostim and eltrombopag have shown high therapeutic activity in primary ITP, but data of efficacy and safety regarding their use in preparation for splenectomy are missing. Thirty-one adult patients, median age 50 years, with corticosteroids and/or IVIG refractory persistent and chronic ITP who were treated with TPO-RAs (romiplostim= 24; eltrombopag= 7) with the aim to increase platelet count and allow a safer execution of splenectomy were retrospectively evaluated. Twenty-four patients (77%) responded to the use of TPO-RAs with a median platelet count that increased from 11 x 109 /L before starting TPO-RAs to 114 x 109 /L pre-splenectomy, but a concomitant treatment with corticosteroids and/or IVIG was required in 19 patients.
4.Severe Thrombotic Complication of Eltrombopag in a Cirrhotic Patient.
Baumann AJ1, Wheeler DS1, Varadi G2, Feyssa E3. ACG Case Rep J. 2016 Jan 20;3(2):121-3. doi: 10.14309/crj.2016.20. eCollection 2016.
We present a patient with hepatitis C virus (HCV) and cirrhosis who was treated with eltrombopag for idiopathic thrombocytopenic purpura and was incidentally found to have a right atrial thrombus with extension into the left internal jugular vein. Eltrombopag was discontinued and the patient was treated with thrombectomy and anticoagulation. Given the proposed use of eltrombopag in HCV-associated thrombocytopenia, we advise caution when treating cirrhotics who are at higher intrinsic risk of thrombosis.