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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., (© 2019 by The American Society of Hematology.)

Problem: There is a recognized need to translate scientific discoveries to patient-oriented clinical research (POCR). Several obstacles interfere with the successful recruitment and retention of physicians for POCR careers., Approach: The American Society of Hematology developed a yearlong educational and mentoring experience, the Clinical Research Training Institute (CRTI), for early-career physician-scientists from multiple institutions throughout the United States and Canada pursuing POCR careers. Several academic outcome measures of the 140 participants in the first seven years (2003-2010) of CRTI were evaluated by reviewing former trainee participants' curriculum vitae and survey responses., Outcomes: Ethnic, racial, and gender diversity of CRTI trainees was reflective of the proportions represented across U.S. hematology/oncology fellowship programs. Eighty-six percent (109/126) of trainees reported success establishing a POCR study; nearly half (62/126) had primarily research-focused jobs. Former CRTI trainees received at least 262 external grant awards and published 1,035 peer-reviewed manuscripts, 173 chapters, and 115 review articles., Next Steps: Because mentorship is key to developing a successful career, the CRTI program is being modified to enhance longitudinal mentorship by CRTI faculty mentors and mentors at trainees' home institutions, as well as to encourage the establishment of collaborations and the potential for research project success. Efforts to make the CRTI experience available to more phy sicians, include more CRTI graduates as faculty, and increase participation by hematologists from backgrounds under represented in medicine are under way.

Background: Incident idiopathic thrombotic thrombocytopenic purpura (TTP) is an uncommon, potentially fatal blood disorder for which there are little or no data on health care costs., Study Design and Methods: Patients satisfying a validated claims-based algorithm including an inpatient diagnosis of TTP and plasma exchange (PE) procedure during the period January 1, 2001 to May 31, 2008 were identified in the HealthCore Integrated Research Database. To characterize patterns of treatment and payments, a quantitative evaluation of comorbidities and treatments, health care utilization, and payments among this population of patients was conducted. All patients were followed until death, end of health plan enrollment, or 365 days after the TTP hospitalization, whichever occurred first., Results: One hundred fifty-one patients met the claims coding algorithm. Mean total health care payments for the TTP hospitalization were $56,347 (standard deviation [SD] $80,230). Ten patients (6.6%) died during the hospitalization for TTP. Mean payments for PE services in the month following discharge were $9127 (SD $20,840). Several patients required prolonged PE during the acute TTP phase (up to 116 separate exchanges over a period of 365 days), prolonging required treatment and skewing payments and resource utilization during the 365-day period following discharge from the index TTP hospitalization., Conclusion: These data document the health care resource utilization by patients with idiopathic TTP, demonstrating that management of these patients is not only expensive but also skewed, with some patients requiring prolonged treatment. These data can contribute to cost-effectiveness models when new treatments for TTP become available., (© 2012 American Association of Blood Banks.)

Drug-induced immune thrombocytopenia (DITP) is often suspected in patients with acute thrombocytopenia unexplained by other causes, but documenting that a drug is the cause of thrombocytopenia can be challenging. To provide a resource for diagnosis of DITP and for drug safety surveillance, we analyzed 3 distinct methods for identifying drugs that may cause thrombocytopenia. (1) Published case reports of DITP have described 253 drugs suspected of causing thrombocytopenia; using defined clinical criteria, 87 (34%) were identified with evidence that the drug caused thrombocytopenia. (2) Serum samples from patients with suspected DITP were tested for 202 drugs; drug-dependent, platelet-reactive antibodies were identified for 67 drugs (33%). (3) The Food and Drug Administration's Adverse Event Reporting System database was searched for drugs associated with thrombocytopenia by use of data mining algorithms; 1444 drugs had at least 1 report associated with thrombocytopenia, and 573 (40%) drugs demonstrated a statistically distinctive reporting association with thrombocytopenia. Among 1468 drugs suspected of causing thrombocytopenia, 102 were evaluated by all 3 methods, and 23 of these 102 drugs had evidence for an association with thrombocytopenia by all 3 methods. Multiple methods, each with a distinct perspective, can contribute to the identification of drugs that can cause thrombocytopenia.

It was the purpose of the present study to validate administrative claims codes for idiopathic thrombotic thrombocytopenic purpura (TTP) in a commercially-insured US population. Patients with at least one medical claim with ICD-9 code 446.6X between 1/1/2001 and 5/31/2008 were identified in the HealthCore Integrated Research Database (HIRD). A chart abstraction form was developed to enable case determination for patients identified by the claims code. Two clinical experts, not involved in the design of the study, reviewed the abstracted medical record data and determined whether definite evidence supporting the diagnosis of TTP was present. The positive predictive value (PPV) of the claims coding algorithm for cases assessed by both reviewers was computed. The claims algorithm was further refined and the PPV of the refined algorithm was computed. One hundred eighty-nine abstracted charts were reviewed by two clinical experts; 86 were assessed to have definite evidence supporting the diagnosis of TTP (PPV 45.5% [86/189; 95% confidence interval (CI), 38.3-52.9%]). Refinement of the claims algorithm first included the use of plasma exchange treatment, resulting in 103 potential cases, of which 67 were assessed to have definite evidence supporting the diagnosis of TTP (PPV 65.0%; 95% CI, 55.0-74.2%). Further refinement of the claims algorithm ruled out alternative diagnoses that may mimic TTP; 34 were assessed to have definite evidence supporting the diagnosis of TTP (PPV 72.3% [34/47; 95% CI, 57.4-84.4%]).Our findings demonstrate the difficulty of confirming the diagnosis of rare disorders that lack definite diagnostic criteria, and indicate that more complex claims coding algorithms are necessary for identifying these disorders.

Reports of the incidence of ITP are few and their methodology is variable. Accurate estimates of the incidence of immune thrombocytopenic purpura (ITP) are important to understand the medical and public health impact of the disease. To critically review all published reports on the incidence of ITP in children and adults, all articles identified on the Medline database (searched January 1, 1966-August 7, 2009) that reported data on the incidence of ITP were retrieved. Articles which directly estimated the incidence of ITP were selected for review. Eight articles reported the incidence of acute ITP in children. After review, four were determined to have the strongest estimates, based on the method of patient identification and study design. The lowest incidence estimate in these four studies was 2.2 per 10(5) children/year (95% confidence interval 1.9, 2.4) and the highest incidence estimate was 5.3 per 10(5) children/year (95% confidence interval 4.3, 6.4). Three studies reported the incidence of ITP in adults. The estimate from the article with the strongest methodology reported an incidence estimate of 3.3 per 10(5) adults/year. The current strongest estimate of the incidence of acute ITP in children is between 1.9 and 6.4 per 10(5) children/year; for adults the current strongest estimate of the incidence of ITP is 3.3 per 10(5) adults/year. An important limitation of these studies is that they are primarily from Europe and may not be generalizable to all regions., (2009 Wiley-Liss, Inc.)

Purpose: To assess physician use of erythropoietin in cancer patients before publication of the American Society of Clinical Oncology/American Society of Hematology guidelines., Methods: Questionnaires about erythropoietin use in practice and 12 hypothetical clinical scenarios involving patients with cancer were mailed to 2000 oncologists/hematologists in the United States and 19 other countries. Response rates were 30% in the United States and 25% internationally. Data on erythropoietin use for ovarian cancer were obtained from one clinical trial. Multivariate regression models assessed predictors of erythropoietin prescription., Results: Most physicians selected a hemoglobin level < or =10 g/dL as an upper threshold for erythropoietin use (36% to 51% of U.S. physicians and 21% to 32% of foreign physicians). Frequent erythropoietin use (defined as use in at least 10% of cancer patients) was higher in the United States than elsewhere (adjusted odds ratio [OR] = 5.8; 95% confidence interval [CI]: 2.5 to 13.4). Among U.S. physicians, those who said they used erythropoietin frequently were more likely to be in fee-for-service than managed care settings (OR = 2.2; 95% CI: 1.3 to 3.7). Those who reported never using erythropoietin practiced in countries that had lower annual per capita health care expenditures, lower proportions of privately funded health care, and a national health service (P <0.05 for all comparisons). Of 235 ovarian cancer patients who received topotecan, 38% (45/118) of U.S. patients and 2% (2/117) of European patients who developed grade 1 anemia (hemoglobin level between 10 and 12 g/dL) were treated with erythropoietin (P <0.01)., Conclusion: Financial considerations and a hemoglobin level <10 g/dL appear to influence erythropoietin use in the United States, whereas financial considerations alone determine erythropoietin use abroad.

The purpose of this study was to update physicians' self-reported initial management practices for childhood idiopathic thrombocytopenic purpura (ITP) from an initial survey in 1997. A questionnaire was sent by e-mail in October 2001 to 753 members of the American Society of Pediatric Hematology/Oncology (ASPH/O). The questionnaire had 14 questions, based on the clinical presentation of a 5-year-old boy with ITP, a platelet count of 7,000/microL, scattered petechiae, and no mucous membrane bleeding. Two hundred eighteen (29%) surveys were returned. In response to questions regarding initial treatment, 33% of physicians said they would always administer drug therapy, 38% usually, 15% sometimes, and 14% rarely/never. When asked which agent would be used if drug treatment were prescribed, 45% reported anti-D, 33% IVIG, 20% prednisone, and 2% other regimens. Only 34% of physicians would always or usually hospitalize such a patient. Hospitalization was more likely if a physician responded that he or she would always or usually use drug therapy. Physicians who saw more ITP patients were more likely to self-report sometimes or rarely/never prescribing drug therapy. Self-reported initial management of ITP by ASPH/O members in 2001 is similar to 1997 regarding the percentage of pediatric hematologists who would not use drug therapy. Among physicians who would use drug treatment, there was an increased use of anti-D and decreased use of IVIG and prednisone. This information provides the basis for designing a randomized clinical trial to compare the effect of different management strategies on the outcomes of bleeding symptoms, side effects of therapy, costs, and quality of life.