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Treatment of chronic idiopathic thrombocytopenic purpura (ITP) varies according to the severity of the disease, and may include only careful observation. ITP involves a tendency to bruise and bleed easily because of abnormally low numbers of blood platelets. ITP may be caused by an autoimmune reaction against platelets, and must be distinguished from thrombocytopenia caused by drugs or underlying disease or thrombocytopenia acquired before birth (congenital). Mild (platelet counts above 50,000/cubic mm) or moderate (counts between 30,000 and 50,000/cubic mm) ITP may not require treatment. Severe, symptomatic ITP is usually treated first with prednisone. Surgical removal of the spleen is also commonly performed. Severe hemorrhage can be treated with platelet transfusion, augmented by intravenous immune globulin. Various experimental drug regimens have been tested with mixed response. Patient age and lifestyle play an important part in determining how aggressively to treat chronic ITP.

Circulating platelets play an important role in limiting bleeding. When a blood vessel is broken, some platelets adhere to the underlying subendothelial matrix. The platelets also aggregate among themselves, and begin the formation of a plug to block the broken vessel. The adhesion of platelets to the subendothelial matrix also results in platelet activation, which leads to the secretion of granules from the platelets and the synthesis of thromboxane A2; these events recruit more platelets into the growing plug. Abnormalities in platelet function are not rare. However, it is often difficult to adequately assess the importance of abnormal platelet function. There are patients with some signs of platelet abnormality, such as common nosebleeds and easy bruising, who are healthy and are found to have normal platelet function on laboratory testing. Conversely, there are patients with abnormal laboratory values, but no symptoms. The situation is complicated by the vast number of everyday substances associated with platelet abnormalities. While the effects of aspirin on platelet function and the risk of bleeding are firmly established, a great many other substances are likely to exert similar effects, including substances in onions, garlic, and Chinese black tree fungus. (In restaurants, this fungus is generally referred to with the more palatable, but less accurate description of 'mushroom'.) The authors provide a detailed description of the antipsychotics, antibiotics, cardiovascular drugs, and chemotherapeutic agents that can produce abnormalities of platelet function. They also provide a detailed description of the role laboratory evaluation of platelet function should play in the evaluation and management of patients. Fortunately, however, in most cases the clinical effects of abnormal platelet function are mild. With the exception of a few surgical procedures in which the consequences of bleeding can be especially dangerous, such as neurosurgical procedures, it is generally not necessary to postpone surgery because of platelet abnormalities. (Consumer Summary produced by Reliance Medical Information, Inc.)

Abstract Background Immune thrombocytopenic purpura (ITP), a condition characterized by autoimmune-mediated platelet destruction and suboptimal platelet production, is associated with symptoms such as bruising, epistaxis, menorrhagia, mucosal bleeding from the gastrointestinal and urinary tracts and, rarely central nervous system bleeding. The aim of this research is to develop a conceptual model to describe the impact of ITP and its treatment on patients' health-related quality of life (HRQoL). Methods A literature search and focus groups with adult ITP patients were conducted to identify areas of HRQoL affected by ITP. Published literature was reviewed to identify key HRQoL issues and existing questionnaires used to assess HRQoL. Focus group transcripts were reviewed, and common themes were extracted by grouping conceptual categories that described the impact on HRQoL. Results The literature synthesis and themes from the focus group data suggest that decreased platelet counts, disease symptoms, and treatment side effects influence multiple domains of HRQoL for ITP patients. Key areas affected by ITP and its treatments include emotional and functional health, work life, social and leisure activities, and reproductive health. Conclusion ITP affects various areas of HRQoL. This conceptual model will help inform the evaluation of therapeutic strategies for ITP.

Abstract Background No validated disease-specific measures are available to assess health-related quality of life (HRQoL) in adult subjects with immune thrombocytopenic purpura (ITP). Therefore, we sought to develop and validate the ITP-Patient Assessment Questionnaire (ITP-PAQ) for adult subjects with ITP. Methods Information from literature reviews, focus groups with subjects, and clinicians were used to develop 50 ITP-PAQ items. Factor analyses were conducted to develop the scale structure and reduce the number of items. The final 44-item ITP-PAQ, which includes ten scales [Symptoms (S), Bother-Physical Health (B), Fatigue/Sleep (FT), Activity (A), Fear (FR), Psychological Health (PH), Work (W), Social Activity (SA), Women's Reproductive Health (RH), and Overall (QoL)], was self-administered to adult ITP subjects at baseline and 7–10 days later. Test-retest reliability, internal consistency reliability, construct and known groups validity of the final ITP-PAQ were evaluated. Results Seventy-three subjects with ITP completed the questionnaire twice. Test-retest reliability, as measured by the intra-class correlation, ranged from 0.52–0.90. Internal consistency reliability was demonstrated with Cronbach's alpha for all scales above the acceptable level of 0.70 (range: 0.71–0.92), except for RH (0.66). Construct validity, assessed by correlating ITP-PAQ scales with established measures (Short Form-36 v.1, SF-36 and Center for Epidemiologic Studies Depression Scale, CES-D), was demonstrated through moderate correlations between the ITP-PAQ SA and SF-36 Social Function scales (r = 0.67), and between ITP-PAQ PH and SF-36 Mental Health Scales (r = 0.63). Moderate to strong inter-scale correlations were reported between ITP-PAQ scales and the CES-D, except for the RH scale. Known groups validity was evaluated by comparing mean scores for groups that differed clinically. Statistically significant differences (p < 0.01) were observed when subjects were categorized by treatment status [S, FT, B, A, PH, and QoL, perceived effectiveness of ITP treatment [S], and time elapsed since ITP diagnosis [PH]. Conclusion Results provide preliminary evidence of the reliability and validity of the ITP-PAQ in adult subjects with ITP. Further work should be conducted to assess the responsiveness and to estimate the minimal clinical important difference of the ITP-PAQ to more fully understand the impact of ITP and its treatments on HRQoL.