scholarly journals Efficacy and Safety of Rituximab for Refractory and Relapsing Thrombotic Thrombocytopenic Purpura: A Cohort of 10 Cases

2015 ◽  
Vol 8 ◽  
pp. CMBD.S25326 ◽  
Author(s):  
Halima El Omri ◽  
Ruba Y. Taha ◽  
Amna Gamil ◽  
Firyal Ibrahim ◽  
Hisham Al Sabah ◽  
...  
Keyword(s):  
Complete Remission ◽  
Thrombotic Thrombocytopenic Purpura ◽  
Dose Range ◽  
Thrombocytopenic Purpura ◽  
Clinical Criteria ◽  
Severe Deficiency ◽  
Life Threatening ◽  
Multiple Relapses

Objective Idiopathic thrombotic thrombocytopenic purpura (TTP) is a life-threatening disorder mediated by autoantibodies directed against ADAMTS13. This provides a rationale for the use of rituximab in this disorder. We report our experience and the outcome of 10 cases of TTP (9 refractory and 1 relapsing) successfully treated with rituximab in combination with plasma exchange (PE) and other immunosuppressive treatments. Methods The diagnosis of TTP was based on clinical criteria and supported by severe deficiency of ADAMTS13 activity and presence of inhibitors in seven cases. Rituximab was started after a median of 18.6 sessions of PE (range: 5-35) at the dose of 375 mg/m2/week for 4-8 weeks. Results Complete remission was achieved in all patients after a median time of 14.4 days of the first dose (range: 6-30). After a median follow-up of 30 months (range: 8-78), eight patients were still in remission and two developed multiple relapses, treated again with the same therapy, and achieved complete responses; they are alive, and in complete remission after a follow-up of 12 and 16 months. Conclusion Rituximab appears to be a safe and effective therapy for refractory and relapsing TTP. However, longer follow-up is recommended to assess relapse and detect possible long-term side effects of this therapy.

scholarly journals How I treat thrombotic thrombocytopenic purpura in pregnancy

Blood ◽  
2020 ◽  
Vol 136 (19) ◽  
pp. 2125-2132
Author(s):  
Barbara Ferrari ◽  
Flora Peyvandi
Keyword(s):  
Differential Diagnosis ◽  
Thrombotic Thrombocytopenic Purpura ◽  
Current Knowledge ◽  
Fetal Outcome ◽  
Subsequent Pregnancy ◽  
Thrombocytopenic Purpura ◽  
Severe Deficiency ◽  
Life Threatening ◽  
High Index Of Suspicion ◽  
In Pregnancy

Abstract Thrombotic thrombocytopenic purpura (TTP) is an acute, life-threatening thrombotic microangiopathy (TMA) caused by acquired or congenital severe deficiency of ADAMTS13. Pregnancy is a recognized risk factor for precipitating acute (first or recurrent) episodes of TTP. Differential diagnosis with other TMAs is particularly difficult when the first TTP event occurs during pregnancy; a high index of suspicion and prompt recognition of TTP are essential for achieving a good maternal and fetal outcome. An accurate distinction between congenital and acquired cases of pregnancy-related TTP is mandatory for safe subsequent pregnancy planning. In this article, we summarize the current knowledge on pregnancy-associated TTP and describe how we manage TTP during pregnancy in our clinical practice.

scholarly journals Open ADAMTS13, induced by antibodies, is a biomarker for subclinical immune-mediated thrombotic thrombocytopenic purpura

Blood ◽  
2020 ◽  
Author(s):  
Elien Roose ◽  
An-Sofie Schelpe ◽  
Edwige Tellier ◽  
György Sinkovits ◽  
Bérangère S Joly ◽  
...  
Keyword(s):  
Thrombotic Thrombocytopenic Purpura ◽  
Adamts13 Activity ◽  
Thrombocytopenic Purpura ◽  
Follow Up Study ◽  
Open Conformation ◽  
Immune Mediated ◽  
Long Term Follow Up ◽  
Donor Plasma

Recently, we showed that during the acute phase of immune-mediated thrombotic thrombocytopenic purpura (iTTP), ADAMTS13 circulates in an open conformation. Although the cause of this conformational change in acute iTTP remains elusive, ADAMTS13 is mainly closed in iTTP patients (i) in remission with an ADAMTS13 activity >50% and undetectable anti-ADAMTS13 autoantibodies, and (ii) after rituximab treatment, suggesting a role for anti-ADAMTS13 autoantibodies. Therefore, IgGs from 18 acute iTTP patients were purified and added to closed ADAMTS13 in healthy donor plasma. This resulted in open ADAMTS13 in 14/18 (78%) samples, proving that indeed anti-ADAMTS13 autoantibodies can induce an open ADAMTS13 conformation. To further elucidate the conformation of ADAMTS13 in iTTP patients, we studied a novel iTTP patient cohort (n=197) that also included plasma samples of iTTP patients in remission where ADAMTS13 activity was <50%. The open ADAMTS13 conformation was not only found during acute iTTP but also in patients in remission with an ADAMTS13 activity <50% and in half of the patients with an ADAMTS13 activity >50%, although free anti-ADAMTS13 autoantibodies were not always detected. Thus open ADAMTS13 is not only a hallmark of acute iTTP, but also a novel biomarker to detect subclinical iTTP in patients in remission. Finally, a long term follow-up study in one iTTP patient showed that the open conformation precedes a severe drop in ADAMTS13 activity. In conclusion, we have shown that anti-ADAMTS13 autoantibodies from iTTP patients induce an open ADAMTS13 conformation. Most importantly, an open ADAMTS13 conformation is a biomarker for subclinical iTTP and could become an important tool in TTP management.

A 14-Year Experience in the Management of Patients with Acquired Immune Thrombotic Thrombocytopenic Purpura in Northern Israel

2015 ◽  
Vol 134 (3) ◽  
pp. 170-176 ◽  
Author(s):  
Nadav Rinott ◽  
Tatiana Mashiach ◽  
Netanel A. Horowitz ◽  
Liliana Schliamser ◽  
Galit Sarig ◽  
...  
Keyword(s):  
Thrombotic Thrombocytopenic Purpura ◽  
Median Number ◽  
Retrospective Evaluation ◽  
Thrombocytopenic Purpura ◽  
Major Benefit ◽  
Life Threatening ◽  
Complicated Course ◽  
Relapse Rates

Acquired idiopathic thrombotic thrombocytopenic purpura (I-TTP) is a life-threatening microangiopathic disorder usually treated with therapeutic plasma exchange (TPE). The current study assessed the role of rituximab in the treatment of complicated I-TTP. The sequence of TTP events was compared in a group of I-TTP patients treated with TPE and a cohort of refractory or relapsed patients who also received rituximab. This retrospective evaluation included 45 I-TTP patients, treated between January 2000 and October 2013, who underwent at least 3 TPE procedures and were followed up until December 2013 or death. Thirty-one patients with an uncomplicated course received TPE only. Fourteen patients had a complicated course due to either a primary refractory/exacerbated disease (n = 5) or relapse (n = 9) and received rituximab together with TPE. The median number of TPE procedures performed in the first TTP episode in the uncomplicated cohort and groups with primary refractory or relapsed TTP was 11, 27 and 45, respectively. The relapse rates per follow-up year in the uncomplicated I-TTP, primary refractory and relapsed I-TTP groups were 0.18, 0.2 and 0.6 episodes, respectively. After rituximab therapy this rate dropped to 0.2 per year in the relapsed subgroup. In conclusion, about a quarter of patients with I-TTP had a complicated course and experienced a major benefit from rituximab in terms of effectiveness and safety.

scholarly journals Preemptive rituximab prevents long-term relapses in immune-mediated thrombotic thrombocytopenic purpura

Blood ◽  
2018 ◽  
Vol 132 (20) ◽  
pp. 2143-2153 ◽  
Author(s):  
Matthieu Jestin ◽  
Ygal Benhamou ◽  
An-Sofie Schelpe ◽  
Elien Roose ◽  
François Provôt ◽  
...  
Keyword(s):  
Thrombotic Thrombocytopenic Purpura ◽  
Relapse Rate ◽  
Thrombocytopenic Purpura ◽  
Immune Mediated ◽  
Key Points ◽  
Long Term Follow Up ◽  
Adamts13 Deficiency ◽  
Risk Balance

Key Points TTP patients who display persistent and severe ADAMTS13 deficiency after remission have a relapse rate of 74% during long-term follow-up. Preemptive rituximab can decrease TTP relapses in 85% of patients with a favorable benefit-risk balance.

The Oklahoma thrombotic thrombocytopenic purpura-haemolytic uraemic syndrome Registry

2013 ◽  
Vol 33 (02) ◽  
pp. 105-112 ◽  
Author(s):  
S. K. Vesely ◽  
D. R. Terrell ◽  
C. C. Deford ◽  
J. A. Reese ◽  
Z. L. Al-Nouri ◽  
...  
Keyword(s):  
Patient Care ◽  
Thrombotic Thrombocytopenic Purpura ◽  
Haemolytic Uraemic Syndrome ◽  
Registry Data ◽  
Thrombocytopenic Purpura ◽  
Research Education ◽  
Long Term Follow Up ◽  
And Function

SummaryThe Oklahoma Thrombotic Thrombocytopenic Purpura-Haemolytic Uraemic Syndrome (TTPHUS) Registry has a 24 year record of success for collaborative clinical research, education, and patient care. This article tells the story of how the Registry began and it describes the Registry’s structure and function. The Registry provides a model for using a cohort of consecutive patients to investigate a rare disorder. Collaboration between Oklahoma, United States and Bern, Switzerland has been the basis for successful interpretation of Registry data.Registry data have provided new insights into the evaluation and management of TTP. Because recovery from acute episodes of TTP has been assumed to be complete, the increased prevalence of hypertension, diabetes, depression, and death documented by long-term follow-up was unexpected. Registry data have provided opportunities for projects for students and trainees, education of physicians and nurses, and also for patients themselves. During our follow-up, patients have also educated Registry investigators about problems that persist after recovery from an acute episode of TTP. Most important, Registry data have resulted in important improvements for patient care.

scholarly journals Long-term, sub-clinical cardiac and renal complications in patients with multiple relapses of thrombotic thrombocytopenic purpura

2010 ◽  
Vol 149 (4) ◽  
pp. 623-625 ◽  
Author(s):  
Srividya Viswanathan ◽  
Brad H. Rovin ◽  
Ganesh B. Shidham ◽  
Subha V. Raman ◽  
Mitchell Weinberg ◽  
...  
Keyword(s):  
Thrombotic Thrombocytopenic Purpura ◽  
Thrombocytopenic Purpura ◽  
Renal Complications ◽  
Multiple Relapses

scholarly journals THROMBOTIC THROMBOCYTOPENIC PURPURA (TTP) AND MODERN APPROACH TO ITS INVESTIGATION AND TREATMENT

2019 ◽  
pp. 12-13
Author(s):  
K. Ukleba ◽  
L. Gvetadze
Keyword(s):  
Thrombotic Thrombocytopenic Purpura ◽  
Severe Thrombocytopenia ◽  
Thrombocytopenic Purpura ◽  
Modern Approach ◽  
Severe Deficiency ◽  
Life Threatening ◽  
Adamts13 Deficiency ◽  
Von Willebrand ◽  
Willebrand Factor

Thrombotic thrombocytopenic purpura (TTP) is a rare and life-threatening thrombotic microangiopathy characterized by microangiopathic hemolytic anemia, severe thrombocytopenia, and organ ischemia linked to disseminated microvascular platelet rich-thrombi. TTP is specifically related to a severe deficiency in ADAMTS13 (a disintegrin and metalloprotease with thrombospondin type 1 repeats, member 13), the specific von Willebrand factor-cleaving protease. ADAMTS13 deficiency is most frequently acquired via ADAMTS13 autoantibodies, but rarely, it is inherited via mutations of the ADAMTS13 gane. The first acute episode of TTP usually occurs during adulthood, with a predominant anti – ADAMTS13 autoimmune etiology. In rare cases, however, TTP begins as soon as childhood, with frequent inherited forms. TTP is 2 – fold more frequent in women, and its outcome is characterized by a relapsing tendency.

Long-term salvage therapy with cyclosporin A in refractory idiopathic thrombocytopenic purpura

Blood ◽  
2002 ◽  
Vol 99 (4) ◽  
pp. 1482-1485 ◽  
Author(s):  
Giovanni Emilia ◽  
Monica Morselli ◽  
Mario Luppi ◽  
Giuseppe Longo ◽  
Roberto Marasca ◽  
...  
Keyword(s):  
Cyclosporin A ◽  
Idiopathic Thrombocytopenic Purpura ◽  
Complete Response ◽  
Term Treatment ◽  
Thrombocytopenic Purpura ◽  
Long Term Treatment ◽  
Life Threatening ◽  
Difficult Challenge

Treatment of severe, chronic idiopathic thrombocytopenic purpura (ITP) refractory to most usual therapies is a difficult challenge. Little information exists on the clinical use of cyclosporin A (CyA) in the treatment of ITP. This report describes long-term treatment with CyA (median, 40 months) and follow-up (median, 36.8 months) in 12 adult patients with resistant ITP. CyA used in relatively low doses (2.5-3 mg/kg of body weight per day) led to a clinical improvement in 10 patients (83.3%). Five had a complete response (41.1%), 4 a complete response to maintenance therapy (33.3%), and one a partial response (8.3%). Two patients had no response. Most patients with a response (60%) had a long-term remission (mean, 28.6 months) after discontinuation of CyA. One patient had a relapse of ITP 4 years after CyA therapy was stopped. Side effects were moderate and transient, even in patients dependent on continued CyA treatment. CyA seems to represent reasonable salvage treatment in severe, potentially life-threatening, refractory ITP.

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