Trial protocol: a multicentre randomised trial of first-line treatment pathways for newly diagnosed immune thrombocytopenia: standard steroid treatment versus combined steroid and mycophenolate. The FLIGHT trial (original) (raw)
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Frontiers in Hematology
PurposeRecent studies suggested that adding other agents to corticosteroids as a first-line treatment for immune thrombocytopenia (ITP) could improve outcomes. However, combination regimens may increase side effects and costs. To determine clinical factors associated with responses to the first-line steroid at 1 month.Materials and methodsWe retrospectively reviewed the medical records of patients with ITP aged ≥ 18 years, who were treated at Rajavithi Hospital between 2012 and 2020. Clinical data, laboratory results, treatment regimens, and responses to therapy were analyzed.ResultsOf the 226 patients, 76.6% were female. The mean age was 46.5 ± 18.1 years, and the median follow-up duration was 40 months. The proportion of chronic ITP was 97.3%. The complete response and response rates to first-line therapy were 65.5% and 88.9%, respectively. The age over 26 years, presentation clinically non-significant bleeding and a difference in platelet count of >50 x 109/L between days 1 an...
Mycophenolate mofetil therapy for severe immune thrombocytopenia
British Journal of Haematology, 2015
Severe immune thrombocytopenia purpura (ITP) presents a clinical challenge. Second-line treatment options are variable without a precise protocol. We present 46 severe ITP patients treated with mycophenolate mofetil (MMF), retrospectively identified from three London teaching hospitals. Data was collected on patient demographics, co-morbidities and previous treatment strategies. Our key interest was whether there was a sustained response in platelet count to MMF. Patients included 27 males and 19 females whose ages ranged from 19 to 93 years old (median 52Á5 years). Twenty-nine had primary ITP and 17 had secondary ITP, a third of whom had viral-associated disease. The standard dose of MMF was 1 g/day. Twenty-four patients (52%) responded with 15 (33%) achieving a complete response. No active viral-associated ITP patients demonstrated a response to MMF, although numbers were small (n = 4). We were not able to demonstrate a difference between responders and non-responders based on gender, age, previous therapies or time since diagnosis of ITP. Three of four previously splenectomized patients responded, two achieving complete response. We conclude that MMF is a useful steroid-sparing immunosuppressant to be considered in the second-line or later treatment of ITP.
A Retrospective Analysis of the Treatment Approach to Immune Thrombocytopenia in the Real World
Cureus, 2019
Introduction Immune thrombocytopenia (ITP) is an acquired cause of thrombocytopenia in both the adult and children populations due to the accelerated destruction of platelets and/or suppressed platelet production. We present a retrospective analysis of a case series of patients in a single teaching institution with the objective of describing the clinical characteristics and different treatment approaches of patients with ITP. Methods A review of electronic health records was performed in the University Hospital Samaritana, Bogota, for inpatients between 2013 and 2016. Data were extracted for the patients with an ITP diagnosis for variables previously chosen and reviewed for descriptive analysis. Results A total of 55 patients were diagnosed with ITP; of these, 67.3% were female and the median age of this group of patients was 48 years. The majority presented with severe thrombocytopenia with 80% of patients having platelets less than 30000/µL. Of the 55 patients with a final diagnosis of ITP, only 54 received dexamethasone, methylprednisolone, or prednisone as the first-line treatment. The increment in platelet count after seven days of treatment was greater in the group treated with dexamethasone. Conclusion The diagnosis of ITP is of exclusion, there is no gold standard test, however, as it was shown in our results, various unnecessary studies are performed that increase costs during the diagnostic approach. Evidence supports that treatment with high-dose dexamethasone is associated with faster short-and greater long-term efficacy as compared to other steroids, however, it is not always the first choice in real-world patients. It is our belief that the implementation of a guideline will reduce testing and costs, and ensure better treatment options for our patient population.
Clinical and Applied Thrombosis/Hemostasis, 2018
Adult immune thrombocytopenia (ITP) commonly relapses after stopping treatments. This may be preventable by low-dose steroids. In this multicenter study, adult patients with ITP who had been responding to corticosteroids were randomized with the 2 strata of newly diagnosed and relapsed ITP to prednisolone 7.5 mg/d or observation for 6 months. Relapses were defined by a platelet count below 30 × 109/L and/or clinical bleeding. There were 75 patients evaluable for the efficacy and 77 for safety. The recurrent ITP comprised 57.3%. During the median follow-up of 42 weeks, there were 20.5% (8/39) and 25% (9/36) of recurrences in the prednisolone and control groups ( P = .643), with the hazard ratio (HR) of 0.75 ( P = .549). The significant factor that could predict recurrences was relapsed ITP with the HR of 2.79 (95% confidence interval, 1.02-7.64, P = .037). Prednisolone showed a trend toward a benefit in the relapsed subgroup ( P = .070). Adverse events were not different ( P = .540) ...
State of the art - how I manage immune thrombocytopenia
British journal of haematology, 2017
The management of patients with immune thrombocytopenia (ITP) is rapidly evolving. Over the last 15 years, a number of novel treatments have improved practice, with many steroid-sparing agents and a reduction in the progression to splenectomy. Although this has improved clinical care, many therapeutic challenges remain. There is no diagnostic test, no biomarkers to direct treatment and few comparative studies to help management decisions. Development of up to date guidelines is difficult with little high-grade evidence. First line treatment continues to be steroids and intravenous immunoglobulins (IVIG) although both are often poorly tolerated and not curative. Common second line treatments include rituximab, immunosuppressive agents, such as azathioprine and mycophenolate mofetil, and the thrombopoietin receptor agonists romiplostim and eltrombopag. There are no comparative studies to decide between these agents and treatment is generally individualized, depending on comorbidity. U...
International Journal of Clinical and Diagnostic Pathology
ITP is considered as 'isolated thrombocytopenia with no clinically apparent associated conditions or other causes of thrombocytopenia' by the American Society for Haematology guidelines. So it is thus a condition that, to a large extent, is a diagnosis of exclusion. Asymptomatic patients of chronic ITP do not require treatment as per the guidelines unless they are bleeding or the platelet count in adults is <30,000/cmm. So here our aim is to retrospectively analyze our data regarding the factors responsible for initiating therapy and the type of therapy instituted based upon the affordability of patients and severity of bleed. We performed an observational study in our Institute in patients diagnosed as chronic ITP from the years Oct 2016 Sept 2019. Out of 113 patients diagnosed as ITP, 17 patients had chronic ITP. Patients with persistent and chronic ITP had a platelet count ranging between 5000/cmm to <1,00,000/cmm. Most common site of bleeding was cutaneous bleed in the form of petechial spots and ecchymosis (15 out of 17 patients). The majority of the patients had steroids as first treatment along with rituximab while second treatment was diverse. Children with asymptomatic thrombocytopenia did not receive any therapy. None of our patients underwent splenectomy. Identification of common clinical presentation and their treatment strategy in our study can helps to that patients who are at increased risk of this disease.
Consensus Paper—ICIS Expert Meeting Basel 2009 treatment milestones in immune thrombocytopenia
Annals of Hematology, 2010
The rarity of severe complications of this disease in children makes randomized clinical trials in immune thrombocytopenia (ITP) unfeasible. Therefore, the current management recommendations for ITP are largely dependent on clinical expertise and observations. As part of its discussions during the Intercontinental Cooperative ITP Study Group Expert Meeting in Basel, the Management working group recommended that the decision to treat an ITP patient be individualized and based mainly on bleeding symptoms and not on the actual platelet count number and should be supported by bleeding scores using a validated assessment tool. The group stressed the need to develop a uniform validated bleeding score system and to explore new measures to evaluate bleeding risk in thrombocytopenic patients-the role of rituximab as a splenectomy-sparing agent in resistant disease was also discussed. Given the apparently high recurrence rate to rituximab therapy in children and the drug's possible toxicity, the group felt that until more data are available, a conservative approach may be considered, reserving rituximab for patients who failed splenectomy. More studies of the effectiveness and side effects of drugs to treat refractory patients, such as TPO mimetics, cyclosporine, mycophenolate mofetil, and cytotoxic agents are required, as are long-term data on postsplenectomy complications. In the patient with either acute or chronic ITP, using a more personalized approach to treatment based on bleeding symptoms rather than platelet count should result in less toxicity and empower both physicians and families to focus on quality-of-life.
Platelets, 2020
Despite the publication in 2009 of a paper on 'terms and definitions of immune thrombocytopenia', (ITP) some unresolved issues remain and are reflected by the disagreement in the treatment suggested for primary ITP in adults. Considering that these disagreements could be ascribed to non-shared goals, a 'consensus' to classify the different lines of treatment for primary ITP in adults according to their indications and goals was proposed in October 2018 to the XIX annual meeting of the Italian Gruppo di Studio delle Piastrine (GSP), a non-profit platelet study group of scientists and physicians. Having approved the project, 60 potential co-authors and experts in the world were invited to take part to a consensus through e-Delphy method and nine of the 12 who initially accepted the invitation completed the work. Agreement was reached on a classification of four lines of treatment for primary ITP in adults based on their indications and goals. The consensus obtained regarded also the criteria, 'timing' included, to consider practicable elective splenectomy in these patients. In our opinion, the classification of the lines of treatment for primary ITP in adults here proposed could facilitate the realization of better shared evidence-based guidelines for the treatment of the disease.
Second-line therapies in immune thrombocytopenia
Hematology, 2016
Immune thrombocytopenia (ITP) is a rare, acquired autoimmune condition characterized by a low platelet count and an increased risk of bleeding. Although many children and adults with ITP will not need therapy beyond historic first-line treatments of observation, steroids, intravenous immunoglobulin (IVIG), and anti-D globulin, others will have an indication for second-line treatment. Selecting a second-line therapy depends on the reason for treatment, which can vary from bleeding to implications for health-related quality of life (HRQoL) to likelihood of remission and patient preference with regard to adverse effects, route of administration, and cost. Published studies of these treatments are limited by lack of comparative trials, in addition to inconsistent outcome measures, definitions, and efficacy endpoints. This article provides an up-to-date comparison of the second-line treatments, highlighting important outcome measures including bleeding, HRQoL, fatigue, and platelet count...