scholarly journals Treatment of thrombotic thrombocytopenic purpura

2021 ◽  
Vol 93 (7) ◽  
pp. 826-829
Author(s):  
Gennadii M. Galstyan ◽  
Alexei A. Maschan ◽  
Elizaveta E. Klebanova ◽  
Irina I. Kalinina
Keyword(s):  
Factor Viii ◽  
Plasma Exchange ◽  
Thrombotic Thrombocytopenic Purpura ◽  
Pediatric Patients ◽  
Treatment Options ◽  
Clotting Factor ◽  
Thrombocytopenic Purpura ◽  
Congenital Thrombotic Thrombocytopenic Purpura

The review discusses approaches to treatment of congenital thrombotic thrombocytopenic purpura (TTP) or Upshaw-Schulman syndrome. In congenital TTP, plasma transfusions are sufficient. Such treatment options as plasma exchange, administration of clotting factor VIII concentrate, recombinant ADAMTS13, are also used. Separately discussed issues of management of patients with TTP during pregnancy, and pediatric patients with TTP.

Relapsed/refractory thrombotic thrombocytopenic purpura treated with N-acetylcysteine: a case report

2018 ◽  
Vol 63 (4) ◽  
pp. 122-124
Author(s):  
Sinan Demircioğlu ◽  
Ömer Ekinci ◽  
Ali Doğan ◽  
Cengiz Demir
Keyword(s):  
Case Report ◽  
Renal Impairment ◽  
Plasma Exchange ◽  
Thrombotic Thrombocytopenic Purpura ◽  
Treatment Options ◽  
Rare Condition ◽  
High Dose ◽  
Thrombocytopenic Purpura ◽  
Loss Of Response ◽  
Refractory Case

Thrombotic thrombocytopenic purpura is a rare condition that presents with microangiopathic haemolytic anaemia, thrombocytopaenia, fever, renal impairment and neurological symptoms. Plasma exchange is a lifesaving treatment for this condition. However, some cases may be non-responsive to plasma exchange, or loss of response may occur. Treatment options for refractory cases include high-dose corticosteroids, rituximab, vincristine, cyclophosphamide, splenectomy, bortezomib and N-acetylcysteine. We present a refractory case of thrombotic thrombocytopenic purpura responding to the last of these therapies.

New treatment options for thrombotic thrombocytopenic purpura

2017 ◽  
Vol 37 (03) ◽  
pp. 211-215 ◽  
Author(s):  
Paul Knöbl
Keyword(s):  
Platelet Aggregation ◽  
Plasma Exchange ◽  
Thrombotic Thrombocytopenic Purpura ◽  
Treatment Options ◽  
Autoimmune Process ◽  
Thrombocytopenic Purpura ◽  
Missing Enzyme ◽  
Disturbed Microcirculation ◽  
Life Threatening ◽  
New Treatment

SummaryThe thrombotic-thrombocytopenic purpura (TTP) is an acute, life-threatening disease, characterised by enhanced platelet aggregation, disturbed microcirculation and organ dysfunction. With the currently available treatment (plasma exchange, infusions, corticosteroids) mortality ist still as high as 10–15 %. Recent, pathophysiology-based developments may improve the outcome. The most promising candidates for future treatment of TTP are: rituximab for termination of the autoimmune process, caplacizumab for prevention of platelet-VWF-interaction, and recombinant ADAMTS13 for replacement of the inhibited or missing enzyme.

scholarly journals Treatment of thrombotic thrombocytopenic purpura

2021 ◽  
Vol 93 (6) ◽  
pp. 736-745
Author(s):  
Gennadii M. Galstyan ◽  
Alexei A. Maschan ◽  
Elizaveta E. Klebanova ◽  
Irina I. Kalinina
Keyword(s):  
Cyclosporin A ◽  
Thrombotic Thrombocytopenic Purpura ◽  
Pediatric Patients ◽  
Treatment Options ◽  
Acute Attack ◽  
Thrombocytopenic Purpura

The review discusses approaches to treatment of acquired thrombotic thrombocytopenic purpuгa (aTTP). In patients with aTTP plasma exchanges, glucocorticosteroids allow to stop an acute attack of TTP, and use of rituximab allows to achieve remission. In recent years, caplacizumab has been used. Treatment options such as cyclosporin A, bortezomib, splenectomy, N-acetylcysteine, recombinant ADAMTS13 are also described. Separately discussed issues of management of patients with TTP during pregnancy, and pediatric patients with TTP.

ADAMTS13 Helps Distinguish Thrombotic Thrombocytopenic Purpura From Other Microangiopathies and Guide Necessity of Plasma Exchange Therapy

Blood ◽  
2011 ◽  
Vol 118 (21) ◽  
pp. 2328-2328
Author(s):  
Neil Shah ◽  
Karen Matevosyan ◽  
James Burner ◽  
Ravindra Sarode
Keyword(s):  
Plasma Exchange ◽  
Thrombotic Thrombocytopenic Purpura ◽  
Conflicts Of Interest ◽  
Genetic Mutations ◽  
Thrombocytopenic Purpura ◽  
Plasma Exchange Therapy ◽  
Severe Deficiency ◽  
Congenital Thrombotic Thrombocytopenic Purpura ◽  
Adamts13 Deficiency ◽  
Clinical Conditions

Abstract Abstract 2328 Introduction: Congenital thrombotic thrombocytopenic purpura (TTP) is caused by genetic mutations in ADAMTS13, a metalloproteinase that cleaves ultra-large VWF multimers to generate normal sized multimers present in circulation. However, acquired deficiency of ADAMTS13 due to auto-antibodies is not universally accepted as diagnostic of idiopathic TTP. Distinguishing idiopathic TTP from other thrombotic microangiopatheis (TMA) is important in guiding treatment as plasma exchange (PE) is only beneficial in TTP and select cases of other microangiopathies such as atypical HUS. Since January 2006 we routinely obtain ADAMTS13 activity performed by FRET assay from The Blood Center of Wisconsin in all patients with TMA. We report the diagnostic utility of ADAMTS13 to distinguish TTP from other forms of TMAs. Methods: A retrospective analysis was performed on consecutive patients with TMA who had ADAMTS13 assayed from January 2006 to October 2010. Demographics, presenting clinical and laboratory features are given in the Table. Responses to therapeutic plasma exchange (TPE), diagnoses at discharge, and other underlying clinical conditions were also recorded. Relevant statistical analysis was performed using unpaired t-test to compare means and Fisher's exact method for contingency tables. Results: We divided our cases based on severe ADAMTS13 deficiency (<10%) as TTP and non-severe deficiency (>20%) as TMA. TMA causes included quinine induced HUS (1), gemcitabine related HUS (1), malignant hypertension/pre-eclampsia/HEELP (3), sepsis (2), MCTD (5), malignancy (3), ITP (1), HIV/opportunistic infection (5), drugs (1), and multiple (5). Conclusions: In our experience severe ADAMTS13 deficiency appears to distinguish TTP from TMA. TMA had better mortality despite either not initiating or discontinuing PE based on ADAMTS13 levels. However, if we had continued PE in TMA then these patients would have been considered as NON-ADAMTS13 deficient TTP who responded well to PE. Disclosures: No relevant conflicts of interest to declare.

scholarly journals Characterization and treatment of congenital thrombotic thrombocytopenic purpura

Blood ◽  
2019 ◽  
Vol 133 (15) ◽  
pp. 1644-1651 ◽  
Author(s):  
Ferras Alwan ◽  
Chiara Vendramin ◽  
Ri Liesner ◽  
Amanda Clark ◽  
William Lester ◽  
...  
Keyword(s):  
Thrombotic Thrombocytopenic Purpura ◽  
Age Of Onset ◽  
Organ Damage ◽  
Compound Heterozygous ◽  
Prophylactic Therapy ◽  
Thrombocytopenic Purpura ◽  
Stroke Incidence ◽  
End Organ Damage ◽  
Congenital Thrombotic Thrombocytopenic Purpura ◽  
Diagnosis Age

Abstract Congenital thrombotic thrombocytopenic purpura (cTTP) is an ultra-rare thrombomicroangiopathy caused by an inherited deficiency of a disintegrin and metalloproteinase with a thrombospondin type 1 motif, member 13 (ADAMTS13). There are limited data on genotype-phenotype correlation; there is no consensus on treatment. We reviewed the largest cohort of cTTP cases, diagnosed in the United Kingdom, over the past 15 years. Seventy-three cases of cTTP were diagnosed, confirmed by genetic analysis. Ninety-three percent were alive at the time of review. Thirty-six percent had homozygous mutations; 64% had compound heterozygous mutations. Two presentation peaks were seen: childhood (median diagnosis age, 3.5 years) and adulthood, typically related to pregnancy (median diagnosis age, 31 years). Genetic mutations differed by age of onset with prespacer mutations more likely to be associated with childhood onset (P = .0011). Sixty-nine percent of adult presentations were associated with pregnancy. Fresh-frozen plasma (FFP) and intermediate purity factor VIII concentrate were used as treatment. Eighty-eight percent of patients with normal blood counts, but with headaches, lethargy, or abdominal pain, reported symptom resolution with prophylactic therapy. The most common currently used regimen of 3-weekly FFP proved insufficient for 70% of patients and weekly or fortnightly infusions were required. Stroke incidence was significantly reduced in patients receiving prophylactic therapy (2% vs 17%; P = .04). Long-term, there is a risk of end-organ damage, seen in 75% of patients with late diagnosis of cTTP. In conclusion, prespacer mutations are associated with earlier development of cTTP symptoms. Prophylactic ADAMTS13 replacement decreases the risk of end-organ damage such as ischemic stroke and resolved previously unrecognized symptoms in patients with nonovert disease.

Sign in / Sign up

Export Citation Format

Share Document