scholarly journals How I treat immune thrombocytopenia: the choice between splenectomy or a medical therapy as a second-line treatment

Blood ◽  
2012 ◽  
Vol 120 (5) ◽  
pp. 960-969 ◽  
Author(s):  
Waleed Ghanima ◽  
Bertrand Godeau ◽  
Douglas B. Cines ◽  
James B. Bussel
Keyword(s):  
Immune Thrombocytopenia ◽  
Second Line ◽  
Cure Rate ◽  
Treatment Needs ◽  
Short Term ◽  
Advantages And Disadvantages ◽  
Thrombopoietin Receptor ◽  
Primary Immune Thrombocytopenia ◽  
Alternative Approaches ◽  
One Year

Abstract The paradigm for managing primary immune thrombocytopenia (ITP) in adults has changed with the advent of rituximab and thrombopoietin receptor agonists (TPO-RAs) as options for second-line therapy. Splenectomy continues to provide the highest cure rate (60%-70% at 5+ years). Nonetheless, splenectomy is invasive, irreversible, associated with postoperative complications, and its outcome is currently unpredictable, leading some physicians and patients toward postponement and use of alternative approaches. An important predicament is the lack of studies comparing second-line options to splenectomy and to each other. Furthermore, some adults will improve spontaneously within 1-2 years. Rituximab has been given to more than 1 million patients worldwide, is generally well tolerated, and its short-term toxicity is acceptable. In adults with ITP, 40% of patients are complete responders at one year and 20% remain responders at 3-5 years. Newer approaches to using rituximab are under study. TPO-RAs induce platelet counts > 50 000/μL in 60%-90% of adults with ITP, are well-tolerated, and show relatively little short-term toxicity. The fraction of TPO-RA–treated patients who will be treatment-free after 12-24 months of therapy is unknown but likely to be low. As each approach has advantages and disadvantages, treatment needs to be individualized, and patient participation in decision-making is paramount.

scholarly journals Second-line administration of thrombopoietin receptor agonists in immune thrombocytopenia: Italian Delphi-based consensus recommendations

2021 ◽  
Vol 12 ◽  
pp. 204062072110483
Author(s):  
Monica Carpenedo ◽  
Erminia Baldacci ◽  
Claudia Baratè ◽  
Alessandra Borchiellini ◽  
Francesco Buccisano ◽  
...  
Keyword(s):  
Platelet Count ◽  
Delphi Technique ◽  
Immune Thrombocytopenia ◽  
Daily Practice ◽  
Second Line ◽  
Thrombopoietin Receptor Agonists ◽  
Receptor Agonists ◽  
Line Therapy ◽  
Thrombopoietin Receptor ◽  
Primary Immune Thrombocytopenia

Introduction: In patients with primary immune thrombocytopenia (ITP), a short course of steroids is routinely given as first-line therapy. However, the response is often transient and additional therapy is usually needed. Thrombopoietin receptor agonists (TPO-RAs) are frequently used as second-line therapy, although there is little clinical guidance on the timing of their administration and on tapering/discontinuation of the drug. To provide clinical recommendations, we used the Delphi technique to obtain consensus for statements regarding administration and on tapering/discontinuation of second-line TPO-RAs among a group of Italian clinicians with expertise in management of ITP. Methods: The Delphi process was used to obtain agreement on five statements regarding initiation and on tapering/discontinuation of second-line TPO-RAs. Agreement was considered when 75% of participants approved the statement. Eleven experts participated in the voting. Results: Full consensus was reached for three of the five statements. The experts held that an early switch from corticosteroids to a TPO-RA has the dual advantage of sparing patients from corticosteroid abuse and improve long-term clinical outcomes. All felt that dose reduction of TPO-RAs can be considered in patients with a stable response and platelet count >100 × 109/L that is maintained for at least 6 months in the absence of concomitant treatments, although there was less agreement in patients with a platelet count >50 × 109/L. Near consensus was reached regarding the statement that early treatment with a TPO-RA is associated with an increase in clinically significant partial or complete response. The experts also agreed that optimization of tapering and discontinuation of TPO-RA therapy in selected patients can improve the quality of life. Conclusion: The present consensus can help to provide guidance on use of TPO-RAs in daily practice in patients with ITP. Plain language summary Second-line administration of thrombopoietin receptor agonists in immune thrombocytopenia There is little guidance on the timing of administration and tapering/discontinuation of thrombopoietin receptor agonists (TPO-RAs) in patients with primary immune thrombocytopenia (ITP). The Delphi technique was used to obtain consensus for five statements. The present consensus among Italian clinicians aims to provide guidance on second-line use of TPO-RAs for patients with ITP in daily practice.

scholarly journals Exposure to Corticoid-Sparing Treatments in Adult Primary Immune Thrombocytopenia before the Chronic Phase in the Era of Thrombopoietin Receptor Agonists in France. a Nationwide Population-Based Study

Blood ◽  
2014 ◽  
Vol 124 (21) ◽  
pp. 2178-2178
Author(s):  
Guillaume Moulis ◽  
Maryse Lapeyre-Mestre ◽  
Jean-Louis Montastruc ◽  
Bertrand Godeau ◽  
Laurent Sailler
Keyword(s):  
Immune Thrombocytopenia ◽  
Age Groups ◽  
Chronic Phase ◽  
Disease Diagnosis ◽  
Second Line ◽  
Thrombopoietin Receptor Agonists ◽  
Receptor Agonists ◽  
Diagnosis Codes ◽  
Thrombopoietin Receptor ◽  
Primary Immune Thrombocytopenia

Abstract Objectives:The aim of this study was to describe the exposure to corticoid-sparing treatments (CST) in adult primary immune thrombocytopenia (ITP) patients during the year before the chronic phase at a nationwide level in the era of thrombopoietin-receptor agonists (TPO-RAs). Methods:Study population was the 2009-2011 cohort of the French Adult Immune Thrombocytopenia: a French pHarmacoepidemiological study (FAITH, n°ENCEPP/SDPP/4574). The FAITH cohort is the cohort of all incident persistent or chronic adult primary ITP patients treated in France, built through the nationwide French health insurance database, named SNIIRAM. It collects prospectively all data regarding hospitalizations, disabling diseases, drug and procedure reimbursements. They are linkable with demographic data. On the 2009-2011 SNIIRAM data, ITP patients were identified with hospital and disabling disease diagnosis codes (D69.3 code of the International Classification of diseases, version-10). The date of diagnosis was refined thanks to out-hospital drug exposures. Secondary ITPs were excluded thanks to diagnosis codes of diseases associated to ITP, searched in the year before and the semester after the diagnosis. We restricted to incident patients, excluding those with a diagnosis during the first semester of the study, and then to patients followed at least 12 months and with at least one exposure to CST during the year after the diagnosis. Exposure to CST was searched through out-hospital dispensing, and during hospital stays for splenectomy, rituximab and polyvalent intravenous immunoglobulins (IVIg). A single dispensing defined exposure, except for IVIg (3 monthly dispensing were mandatory to differentiate IVIg used as CST from acute exposure to treat a serious bleeding). We described the percentage of patients exposed to the distinct CST at least once during the year after ITP diagnosis and detailed the lines of therapy. We compared the patients aged <65 years and those ≥65 years. Results:Out of 2334 adult incident primary ITPs, 1556 were persistent or chronic. Among them, 443 patients were followed at least one year after ITP diagnosis and were exposed to at least one CST during this period. Mean age was 52.7 ± 20.8 years and 59.1% of the patients were females. Patients aged ≥65 years had more frequently mucosal or internal bleeding at ITP onset, and had a higher Charlson’s comorbidity score. Mean time from diagnosis to CST was 3.5 months. CSTs used in >10% of the patients at any time during the year after ITP diagnosis were, by decreasing frequency: rituximab (57.8%), splenectomy (22.1%), TPO-RAs (16.8%), IVIg (15.0%), danazol (14.4%) and dapsone (10.8%). Hydroxychloroquine was used in 6.5% of the patients and immunosupressant (azathioprine, mycophenolate or ciclosporin) in 5.9%. The CST the most frequently used was rituximab in both age groups. Splenectomy was more frequently used in patients aged less than 65 years (25.2% versus 16.4%, p=0.03). In contrast, TPO-RAs and dapsone were more frequently used in patients aged over 65 years (respectively, 24.8% versus 12.8%, p=0.01, and 17.6% versus 7.2%, p=0.0008). The mean number of lines of therapy after corticosteroids was 1.5 (extremes: 1-6) and was not different between the two age groups. As regards CSTs used in second line after corticosteroids, rituximab was the most used (45.4%), followed by IVIg (12.0%), splenectomy (11.3%), danazol (10.1%), dapsone (7.9%) and TPO-RAs (6.3%). There were discrepancies according to the age groups: 11.8% of the patients aged ≥65 years had been exposed to TPO-RAs as second-line compared with 3.5% of younger patients (p=0.0006). Similarly, dapsone was more frequently used as second-line treatment in older patients (13.8% versus 5.2%, p=0.0003), while splenectomy was more frequently used in patients aged <65 years (13.8% versus 6.5%, p=0.02). When detailing the successive lines of therapy, the use of rituximab decreased, while the use of splenectomy and TPO-RA increased in both age groups. Conclusions: Rituximab was the leading CST used as second and third line of therapy in adult primary ITP before the chronic phase in France, whatever the age group. TPO-RAs have modified treatment strategy of persistent primary ITP as soon as they have been marketed. They were mainly used in accordance with their labeling (after two lines of treatment including corticosteroids). Disclosures No relevant conflicts of interest to declare.

How I Manage cyclic thrombocytopenia

Blood ◽  
2020 ◽  
Author(s):  
Paul A Kyrle ◽  
Sabine Eichinger
Keyword(s):  
Platelet Count ◽  
Immune Thrombocytopenia ◽  
Hematological Malignancies ◽  
Severe Bleeding ◽  
Typical Pattern ◽  
Periodic Fluctuation ◽  
Thrombopoietin Receptor ◽  
Primary Immune Thrombocytopenia ◽  
Platelet Count Monitoring ◽  
Mucocutaneous Bleeding

Cyclic thrombocytopenia (CTP) is a rare disease, which is characterized by periodic fluctuation of the platelet count. The pathogenesis of CTP is unknown and most likely heterogeneous. Patients with CTP are almost always misdiagnosed as having primary immune thrombocytopenia (ITP). The interval between ITP and CTP diagnosis can be many years. CTP patients often receive ITP-specific therapies including corticosteroids, thrombopoietin receptor agonists, rituximab and splenectomy which are followed by a transient increase in platelet count that is wrongly attributed to treatment effect with inevitable "relapse". CTP can be diagnosed by frequent platelet count monitoring which reveals a typical pattern of periodic platelet cycling. An early diagnosis of CTP will prevent these patients from being exposed to possibly harmful therapies. The bleeding phenotype is usually mild and consists of mucocutaneous bleeding at the time when the platelet count is at its nadir. Severe bleeding from other sites can occur but is rare. Some patients respond to cyclosporine A or to danazol, but most patients do not respond to any therapy. CTP can be associated with hematological malignancies or disorders of the thyroid gland. Nevertheless, spontaneous remissions can occur, even after many years.

scholarly journals Sustained Remission in Patients with Primary Immune Thrombocytopenia after Romiplostim Tapering and Discontinuation: A Case Series in Real Life Management in Spain

2017 ◽  
Vol 2017 ◽  
pp. 1-8 ◽  
Author(s):  
María-Eva Mingot-Castellano ◽  
Carlos Grande-García ◽  
David Valcárcel-Ferreiras ◽  
Clara Conill-Cortés ◽  
Loreto de Olivar-Oliver
Keyword(s):  
Clinical Practice ◽  
Predictive Factors ◽  
Immune Thrombocytopenia ◽  
Real Life ◽  
Case Series ◽  
Sustained Remission ◽  
Platelet Counts ◽  
Thrombopoietin Receptor ◽  
Primary Immune Thrombocytopenia ◽  
Effective Option

Romiplostim, a thrombopoietin-receptor agonist (TPO-ra), is a highly effective option in primary immune thrombocytopenia (ITP), with 80–90% of patients achieving platelet responses after few weeks of treatment. The evidence showing remissions, that is, sustained platelet counts after romiplostim discontinuation, in patients with ITP refractory to immunosuppressive therapy is steadily increasing. However, there is a lack of guidelines or recommendations addressing how and when to taper romiplostim in clinical practice in patients maintaining elevated and stable platelet counts. Furthermore, given the high heterogeneity of ITP patients, no associated predictive factors have been currently identified. Here, we present 4 representative clinical cases of the daily clinical practice in Spain comprising newly diagnosed, persistent, and both splenectomized and nonsplenectomized chronic ITP patients treated with romiplostim, achieving and maintaining clinical remission (platelet count ≥ 50×109/L for 24 consecutive weeks in the absence of any treatment for ITP) after treatment tapering and discontinuation, without observed safety concerns. Prospective studies identifying clinical and biological predictive factors of sustained response are warranted.

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