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Blood Advances Dec 2019Despite an increase in the number of therapies available to treat patients with immune thrombocytopenia (ITP), there are minimal data from randomized trials to assist...
BACKGROUND
Despite an increase in the number of therapies available to treat patients with immune thrombocytopenia (ITP), there are minimal data from randomized trials to assist physicians with the management of patients.
OBJECTIVE
These evidence-based guidelines of the American Society of Hematology (ASH) are intended to support patients, clinicians, and other health care professionals in their decisions about the management of ITP.
METHODS
In 2015, ASH formed a multidisciplinary guideline panel that included 8 adult clinical experts, 5 pediatric clinical experts, 2 methodologists with expertise in ITP, and 2 patient representatives. The panel was balanced to minimize potential bias from conflicts of interest. The panel reviewed the ASH 2011 guideline recommendations and prioritized questions. The panel used the Grading of Recommendations Assessment, Development and Evaluation (GRADE) approach, including evidence-to-decision frameworks, to appraise evidence (up to May 2017) and formulate recommendations.
RESULTS
The panel agreed on 21 recommendations covering management of ITP in adults and children with newly diagnosed, persistent, and chronic disease refractory to first-line therapy who have non-life-threatening bleeding. Management approaches included: observation, corticosteroids, IV immunoglobulin, anti-D immunoglobulin, rituximab, splenectomy, and thrombopoietin receptor agonists.
CONCLUSIONS
There was a lack of evidence to support strong recommendations for various management approaches. In general, strategies that avoided medication side effects were favored. A large focus was placed on shared decision-making, especially with regard to second-line therapy. Future research should apply standard corticosteroid-dosing regimens, report patient-reported outcomes, and include cost-analysis evaluations.
Topics: Hematology; History, 21st Century; Humans; Purpura, Thrombocytopenic, Idiopathic; United States
PubMed: 31794604
DOI: 10.1182/bloodadvances.2019000966 -
Blood Advances Nov 2019Over the last decade, there have been numerous developments and changes in treatment practices for the management of patients with immune thrombocytopenia (ITP). This...
Over the last decade, there have been numerous developments and changes in treatment practices for the management of patients with immune thrombocytopenia (ITP). This article is an update of the International Consensus Report published in 2010. A critical review was performed to identify all relevant articles published between 2009 and 2018. An expert panel screened, reviewed, and graded the studies and formulated the updated consensus recommendations based on the new data. The final document provides consensus recommendations on the diagnosis and management of ITP in adults, during pregnancy, and in children, as well as quality-of-life considerations.
Topics: Clinical Decision-Making; Combined Modality Therapy; Decision Trees; Disease Management; Humans; Purpura, Thrombocytopenic, Idiopathic; Severity of Illness Index; Treatment Outcome
PubMed: 31770441
DOI: 10.1182/bloodadvances.2019000812 -
Journal of Hematology & Oncology Jan 2023Primary immune thrombocytopenia (ITP) is an immune-mediated bleeding disorder characterized by decreased platelet counts and an increased risk of bleeding. Multiple... (Review)
Review
Primary immune thrombocytopenia (ITP) is an immune-mediated bleeding disorder characterized by decreased platelet counts and an increased risk of bleeding. Multiple humoral and cellular immune abnormalities result in accelerated platelet destruction and suppressed platelet production in ITP. The diagnosis remains a clinical exclusion of other causes of thrombocytopenia. Treatment is not required except for patients with active bleeding, severe thrombocytopenia, or cases in need of invasive procedures. Corticosteroids, intravenous immunoglobulin, and anti-RhD immunoglobulin are the classical initial treatments for newly diagnosed ITP in adults, but these agents generally cannot induce a long-term response in most patients. Subsequent treatments for patients who fail the initial therapy include thrombopoietic agents, rituximab, fostamatinib, splenectomy, and several older immunosuppressive agents. Other potential therapeutic agents, such as inhibitors of Bruton's tyrosine kinase and neonatal Fc receptor, are currently under clinical evaluation. An optimized treatment strategy should aim at elevating the platelet counts to a safety level with minimal toxicity and improving patient health-related quality of life, and always needs to be tailored to the patients and disease phases. In this review, we address the concepts of adult ITP diagnosis and management and provide a comprehensive overview of current therapeutic strategies under general and specific situations.
Topics: Adult; Humans; Blood Platelets; Platelet Count; Purpura, Thrombocytopenic, Idiopathic; Quality of Life; Thrombocytopenia
PubMed: 36658588
DOI: 10.1186/s13045-023-01401-z -
Current Pediatric Reviews 2020Henoch-Schönlein purpura (HSP) is an IgA-mediated systemic smallvessel vasculitis with a predilection for the skin, gastrointestinal tract, joints, and kidneys. It is... (Review)
Review
BACKGROUND
Henoch-Schönlein purpura (HSP) is an IgA-mediated systemic smallvessel vasculitis with a predilection for the skin, gastrointestinal tract, joints, and kidneys. It is the most common form of systemic vasculitis in children.
OBJECTIVE
The study aimed to familiarize physicians with the etiopathogenesis, clinical manifestations, evaluation, and management of children with Henoch-Schönlein purpura.
METHODS
A PubMed search was conducted in January 2020 in Clinical Queries using the key terms "Henoch-Schönlein purpura" OR "IgA vasculitis" OR "anaphylactoid purpura". The search strategy included meta-analyses, randomized controlled trials, clinical trials, observational studies, and reviews published within the past 10 years. Only papers published in the English literature were included in this review. This paper is based on, but not limited to, the search results.
RESULTS
Globally, the incidence of HSP is 10 to 20 cases per 100, 000 children per year. Approximately 90% of cases occur in children between 2 and 10 years of age, with a peak incidence at 4 to 7 years. The diagnosis should be based on the finding of palpable purpura in the presence of at least one of the following criteria, namely, diffuse abdominal pain, arthritis or arthralgia, renal involvement (hematuria and/or proteinuria), and a biopsy showing predominant IgA deposition. Most cases are self-limited. The average duration of the disease is 4 weeks. Long-term complications are rare and include persistent hypertension and end-stage kidney disease. Therapy consists of general and supportive measures as well as treatment of the sequelae of the vasculitis. Current evidence does not support the universal treatment of HSP patients with corticosteroids. Oral corticosteroids may be considered for HSP patients with severe gastrointestinal pain and gastrointestinal hemorrhage.
CONCLUSION
Most cases of HSP have an excellent outcome, with renal involvement being the most important prognostic factor in determining morbidity and mortality. Unfortunately, early steroid treatment does not reduce the incidence and severity of nephropathy in children with HSP. In HSP children who have severe nephritis or renal involvement with proteinuria of greater than 3 months, an angiotensin-converting enzyme inhibitor or angiotensin receptor blocker should be considered in addition to corticosteroids to prevent and/or limit secondary glomerular injury.
Topics: Child; Child, Preschool; Humans; IgA Vasculitis; Kidney; Skin
PubMed: 32384035
DOI: 10.2174/1573396316666200508104708 -
The New England Journal of Medicine Sep 2021
Topics: Autoimmunity; Humans; Purpura, Thrombocytopenic, Idiopathic; Thrombocytopenia
PubMed: 34469652
DOI: 10.1056/NEJMe2110953 -
Hamostaseologie Aug 2019Immune thrombocytopenia (ITP) occurs with an incidence rate of 1.6 to 3.9 per 100,000 patient-years, which increases with age and has a slight female preponderance. ITP... (Comparative Study)
Comparative Study Review
Immune thrombocytopenia (ITP) occurs with an incidence rate of 1.6 to 3.9 per 100,000 patient-years, which increases with age and has a slight female preponderance. ITP is termed acute, persistent or chronic when its duration is <3 months, 3 to 12 months and >12 months, respectively. In this narrative review, we discuss the clinical manifestations of ITP in children and adults. ITP is asymptomatic in some patients; however, when present, bleeding is the most common symptom and can be mild as in petechiae, purpura and epistaxis, or severe and even life threatening in cases of intracranial haemorrhage, and massive gastrointestinal or urinary tract bleeding. A platelet count <10-20 × 10/L, advanced age and prior minor bleeding are risk factors for major bleeding, which is also more common in the months following a new diagnosis of acute ITP. Fatigue and impaired health-related quality of life are also manifestations of ITP that often improve with treatment and improvement in platelet counts. Over long-term follow-up, ITP is also associated with an increased risk of venous and arterial thromboembolism, haematologic malignancy and overall mortality than the general population.
Topics: Adolescent; Adult; Aged; Autoimmune Diseases; Child; Child, Preschool; Fatigue; Female; Follow-Up Studies; Hematologic Neoplasms; Hemorrhage; Humans; Incidence; Male; Middle Aged; Platelet Count; Purpura, Thrombocytopenic, Idiopathic; Quality of Life; Receptors, Thrombopoietin; Risk Factors; Splenectomy; Venous Thromboembolism
PubMed: 30868551
DOI: 10.1055/s-0039-1683416 -
Blood Reviews Sep 2022Thrombotic thrombocytopenic purpura is an acute life-threatening disorder, associated with a mortality of 90% if unrecognised and untreated. The hallmark is... (Review)
Review
Thrombotic thrombocytopenic purpura is an acute life-threatening disorder, associated with a mortality of 90% if unrecognised and untreated. The hallmark is thrombocytopenia and microangiopathic hemolytic anemia, with a blood film characterised by fragmented red cells and end organ damage. The mainstay of treatment is ADAMTS13 replacement, currently with plasma exchange (PEX) and immunosuppression. High dose steroids are used from presentation and anti-CD20 monoclonal antibody therapy, specifically rituximab, is initiated early in the acute disease pathway. The use of the nanobody caplacizumab on confirmation of TTP, by severe ADAMTS13 deficiency (<10iu/dL), has revolutionised acute patient care. Caplacizumab binds the A1 domain, the site on VWF normally occupied by platelets. This results in a quicker normalisation of the platelet count, prevention of exacerbations and refractory disease, reduced PEX and inpatient stay. There is a significant risk of relapse and monitoring of patients allows prophylactic rituximab to be given to prevent further acute admissions.
Topics: ADAMTS13 Protein; Acute Disease; Humans; Plasma Exchange; Platelet Count; Purpura, Thrombotic Thrombocytopenic; Rituximab
PubMed: 35216839
DOI: 10.1016/j.blre.2022.100945 -
Hematology. American Society of... Dec 2022Hematologists are often consulted for thrombocytopenia in pregnancy, especially when there is a concern for a non-pregnancy-specific etiology or an insufficient platelet... (Review)
Review
Hematologists are often consulted for thrombocytopenia in pregnancy, especially when there is a concern for a non-pregnancy-specific etiology or an insufficient platelet count for the hemostatic challenges of delivery. The severity of thrombocytopenia and trimester of onset can help guide the differential diagnosis. Hematologists need to be aware of the typical signs of preeclampsia with severe features and other hypertensive disorders of pregnancy to help distinguish these conditions, which typically resolve with delivery, from other thrombotic microangiopathies (TMAs) (eg, thrombotic thrombocytopenic purpura or complement-mediated TMA). Patients with chronic thrombocytopenic conditions, such as immune thrombocytopenia, should receive counseling on the safety and efficacy of various medications during pregnancy. The management of pregnant patients with chronic immune thrombocytopenia who are refractory to first-line treatments is an area that warrants further research. This review uses a case-based approach to discuss recent updates in diagnosing and managing thrombocytopenia in pregnancy.
Topics: Female; Pregnancy; Humans; Purpura, Thrombocytopenic, Idiopathic; Pregnancy Complications, Hematologic; Purpura, Thrombotic Thrombocytopenic; Thrombotic Microangiopathies; Platelet Count
PubMed: 36485110
DOI: 10.1182/hematology.2022000375 -
Journal of the American Academy of... Apr 2020Retiform purpura is a specific morphology within the spectrum of reticulate eruptions of vascular origin. It develops when blood vessels serving the skin are compromised... (Review)
Review
Retiform purpura is a specific morphology within the spectrum of reticulate eruptions of vascular origin. It develops when blood vessels serving the skin are compromised resulting in downstream cutaneous ischemia, purpura, and necrosis. Identifying retiform purpura is important particularly in the acutely ill patient. It may elucidate the underlying diagnosis, provide prognostic information, and suggest a treatment approach. The differential diagnosis of retiform purpura is vast, reflecting the myriad conditions that can lead to cutaneous vessel wall damage or lumen occlusion. In this article, we give an overview of the differential diagnosis of this cutaneous morphology, provide an approach to workup, and highlight updates in treatment of some of the more common conditions that manifest as retiform purpura.
Topics: Biopsy; Clinical Laboratory Techniques; Diagnosis, Differential; Humans; Medical History Taking; Physical Examination; Purpura; Skin Diseases, Vascular
PubMed: 31479689
DOI: 10.1016/j.jaad.2019.07.112 -
Journal of Thrombosis and Haemostasis :... Oct 2020Despite an increase in our understandings of pathogenesis of thrombotic thrombocytopenic purpura (TTP), the approaches for initial diagnosis and management of TTP vary...
BACKGROUND
Despite an increase in our understandings of pathogenesis of thrombotic thrombocytopenic purpura (TTP), the approaches for initial diagnosis and management of TTP vary significantly.
OBJECTIVE
The evidence-based guidelines of the International Society on Thrombosis and Haemostasis (ISTH) are intended to support patients, clinicians, and other health care professionals in their decisions about the initial diagnosis and management of acute TTP.
METHODS
In June 2018, ISTH formed a multidisciplinary panel that included hematologists, an intensive care physician, nephrologist, clinical pathologist, biostatistician, and patient representatives, as well as a methodology team from McMaster University. The panel composition was designed to minimize the potential conflicts of interests. The panel used the Grading of Recommendations Assessment, Development, and Evaluation approach and the Population, Intervention, Comparison, Outcome framework to develop and grade their recommendations. Public comments were sought and incorporated in the final document.
RESULTS
The panel agreed on three recommendations covering the initial diagnosis with emphasis on the importance of ADAMTS13 testing (eg, activity, anti-ADAMTS13 IgG or inhibitor) and assessment of the pretest probability of TTP by clinical assessment and/or the risk assessment models like the PLASMIC or French score. The panel noted how availability and turnaround time of ADAMTS13 test results might affect early diagnosis and management, in particular the use of caplacizumab.
CONCLUSIONS
There is a lack of high-quality evidence to support strong recommendations for the initial diagnosis and management of a suspected TTP. The panel emphasized the importance of obtaining ADAMTS13 testing in a proper clinical context. Future research should focus on how to monitor and act on ADAMTS13 levels during remission.
Topics: ADAMTS13 Protein; Early Diagnosis; Hemostasis; Humans; Plasma Exchange; Purpura, Thrombotic Thrombocytopenic; Thrombosis
PubMed: 32914582
DOI: 10.1111/jth.15006