Immune thrombocytopenia (ITP), an acquired autoimmune disorder associated with low platelet counts, develops in two to five per 100,000 persons in the United States. The American Society of Hematology recently updated its guidelines on ITP, resulting in 21 recommendations for adults and children with newly diagnosed disease and persistent and chronic disease refractory to first-line therapy in the presence of non–life-threatening bleeding.
The GRADE (Grading of Recommendations, Assessment, Development and Evaluation) approach was utilized. Many of these recommendations lacked evidence to support their strength. When the strength of the recommendation was strong, the guidelines recommended a particular intervention. When the strength of the evidence was conditional, the guidelines only suggested an intervention.
The recommendations for adults with newly diagnosed ITP who are asymptomatic or who have minor mucocutaneous bleeding include the following:
• If the platelet count is <30 x 109/L, the use of corticosteroids over observation is suggested (Recommendation 1a)
• If the platelet count is >30 x 109/L, observation is recommended, and corticosteroids should not be used (Recommendation 1b)
• If the platelet count is <20 X 109/L, hospitalization rather than outpatient management is suggested; however, if an established diagnosis of ITP is present, outpatient management is suggested over hospitalization (Recommendation 2a)
The recommendations for adults with ITP who are corticosteroid-dependent for >3 months or who are corticosteroid-nonresponsive includes the following:
• The use of eltrombopag or romiplostim is suggested if the patient is to be treated with a thrombopoietin receptor agonist (TPO-RA) (Recommendation 6)
• Splenectomy or a TPO-RA is suggested as second-line therapy (Recommendation 7)
• The use of rituximab is suggested rather than splenectomy as second-line therapy (Recommendation 8)
• The recommendations for children with newly diagnosed ITP who have no or only minor bleeding (skin manifestations) include the following:
• If the platelet count is <20 x 109/L, the guidelines suggest against hospitalization and prefer outpatient management (Recommendation 10a)
• If the platelet count is >20 x 109/L, outpatient management is preferred to hospitalization (Recommendation 10b)
• Observation is suggested over glucocorticosteroid use (Recommendation 11)
Among the recommendations for children with ITP who do not respond to first-line treatment, second-line therapies include splenectomy, TPO-RA, and rituximab. Specific recommendations are as follows:
• For children with ITP with non–life-threatening mucosal bleeding and/or diminished health-related quality of life who have not responded to first-line therapy, TPO-RAs is suggested over the use of rituximab (Recommendation 19)
• In this population, TPO-RAs is suggested over splenectomy (Recommendation 20)
• Also, in this population, rituximab is suggested rather than splenectomy (Recommendation 21)
Other treatments for adults and children with ITP include azathioprine, cyclophosphamide, cyclosporine A, danazol, dapsone, mycophenolate, and vinca alkaloids.
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