BackgroundMany patients with immune thrombocytopenia (ITP) may require special attention and long-term treatment. Little is known on the efficacy and tolerability of the drugs used in practice.Material and methodsWe retrospectively reviewed the results of therapy of 400 patients with chronic ITP. All Patients were treated at our institution between 1996–2016 under consideration of guidelines, general recommendations, and individual aspects, including gender, age, weight, comorbidity, patient’s medical history and bleeding risk.ResultsTreatment was not required in 25% of patients (n = 100) during observation. In treated patients (n = 300), the rate of patients that responded and tolerated treatment with prednisolone was 59% (52/88), with azathioprine 32% (29/90), with eltrombopag 49% (31/63), with romiplostim 59% 27/45, with IVIG (intravenous immunoglobulines) 75% (94/126), with anti-D 37% (19/52) and with dexamethasone 60% (25/42) patients. Eighteen treated patients (6%) entered sustained remission after treatment with various drugs. Twenty-six patients underwent splenectomy (Splx) resulting in sustained remission in 15 cases (60%). Only two patients remained refractory to Splx and to all used drugs.DiscussionNone of the currently available drugs used in the treatment of ITP are invariably safe and effective. Responses, the duration of response, intolerability, and the course of disease are unpredictable. Although the treatment of ITP has considerably improved in the recent years, the currently available drugs may rarely cure affected patients. The need for safe and effective therapy in ITP is evident. Optimal treatment decisions for each patient remains a challenge in many cases.

Background: Eltrombopag and romiplostim are thrombopoietin receptor agonists (TPOs) that have been increasingly used for the treatment of immune thrombocytopenia (ITP). Based on our experience, the incidence of abortive treatment with these drugs and the occurrence of adverse reactions that lead to therapy break-off despite response are higher than has been previously suggested. Methods: During the last 8 years, a total of 65 patients were treated with eltrombopag and/or romiplostim at our institute. Results: 36 of a total of 58 patients responded well to eltrombopag. In 12 patients that responded, treatment with eltrombopag was discontinued due to the development of adverse reactions. Eltrombopag was replaced by romiplostim in 23 cases (14 non-responders, 9 patients with adverse reactions). Of these patients, 83% responded to romiplostim. Among all patients treated with romiplostim (n = 32), 75% initially responded; however, 8 of these patients developed adverse reactions. Romiplostim was replaced by eltrombopag in 5 cases (4 due to adverse reactions, 1 non-responsive patient), and only 3 (60%) of these patients were observed to respond to eltrombopag. Conclusion: TPOs often remain ineffective in ITP or result in adverse reactions, which lead to treatment stop or to drug switch. Therefore, alternative treatment options are required.

From a clinical perspective, the onset of therapeutic effects of intravenous immunoglobulin in patients with ITP may occur at an earlier stage and be superior to that previously expected. Failure to measure an increase in platelets in the circulation of 'non-responders' may be explained by an increased consumption of platelets due to recognizable or unrecognizable bleeding in such affected patients.

Background: Dependent on the absence or presence of associated diseases, autoimmune thrombocytopenia (ITP) can be classified as primary or secondary form. The manifestation of the associated diseases is not temporally defined and may occur during observation. Thus the question which disease is the primary one remains unanswered. Methods: All 386 patients included in this study were treated by a single primary physician between 1996 and 2015 at the Charité Berlin and met current ITP criteria. Medical records and investigations were reviewed to assess diseases associated with ITP. Results: Initially, the vast majority of patients presented with primary ITP (isolated disease). Based on our findings, ITP was found to be associated with other abnormalities in most cases. These abnormalities included: positive direct antiglobulin test in 49 of 386 tested patients (13%), affections of the thyroid gland in 41 of 386 tested patients (11%), infections in 30 (8%), solid malignancies in 20 (5%) and hematological malignancies in 10 patients (3%), as well as many other miscellaneous diseases. Moreover, of 160 patients who did not receive prior intravenous immunoglobulin treatment, 40 (25%) showed antibody deficiency. Conclusion: In conclusion, the incidence of ‘true' ITP as a primary disease is less common than has yet been suggested. Additionally, there is evidence that ITP itself predispose affected subjects toward development of other diseases.

The thrombopoietin receptor agonists (TPO-RAs), romiplostim and eltrombopag, stimulate megakaryopoiesis and thereby increase platelet counts. Both drugs are increasingly used in the treatment of immune thrombocytopenic purpura (ITP). To assess the effect of TPO-RAs on trilineage haematopoiesis, colony-forming cell (CFC) assays were performed on peripheral blood mononuclear cells of 8 healthy donors and 52 ITP patients. Additionally, we revaluated the regular and complete blood counts (CBCs) performed during romiplostim therapy in 45 patients and the CBCs performed in 9 patients during eltrombopag therapy. The clonogenic capacity of PBMCs was significantly increased in patients treated with TPO-RAs compared with healthy donors and untreated patients [BFU-E, 69 ± 47; CFU-GM, 61 ± 48; CFU-GEMM, 16 ± 11; CFU-total, 145 ± 94; P values < 0.05)]. Relative leukocytosis was observed in routine blood counts in 12 of 45 (26%) patients treated with romiplostim. The regular CBCs, performed time dependent within the first 5 days, revealed a maximum increase of leukocytes on days 2 and 3 following romiplostim administration. There were no significant changes in red blood cell parameters. None of the affected patients did recognise any significant symptom, which may be related to leukocytosis. Similarly, we observed a statistically significant increase of leukocyte count in a small cohort of ITP patients (n = 9) in whom CBCs were controlled following treatment initiation (P = 0.044). Our results indicate that TPO-RAs may also mobilize haematopoietic stem and progenitor cells (HSPCs) in peripheral blood and the occurrence of such relative leukocytosis may signalize an early symptom of myelofibrosis due to treatment with TPO-RAs.