Pediatric Clinics of North America - CIPERJ

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Pediatr Clin N Am 55 (2008) 287–304 Decision Analysis in Pediatric Hematology Sarah H. O’Brien, MD, MSc a,b, * a Center for Innovation in Pediatric Practice, The Research Institute at Nationwide Children’s Hospital, 700 Children’s Drive, Columbus, OH 43205, USA b Division of Pediatric Hematology/Oncology, Nationwide Children’s Hospital/The Ohio State University, 700 Children’s Drive, Columbus, OH 43205, USA Every day, physicians must make decisions regarding which medical interventions benefit their patients most. For example, a 5-year-old healthy girl presents to an emergency department with a 1-day history of bruising and petechiae. History, physical examination, and initial laboratory studies all are consistent with a diagnosis of immune thrombocytopenic purpura (ITP). The hematologist on call has several decisions to make. Should the child be admitted to the hospital or discharged home Should the child receive therapy or simply be observed If the child receives therapy, should it be immune globulin, anti-D, or steroids There are no results of large clinical trials available to guide or provide definitive answers to any one of these questions. Instead, the hematologist must weigh the risks and benefits of each potential management strategy and choose a course of action that he or she believes is ‘‘the right thing to do.’’ Performing large randomized controlled trials to answer every clinical question in medicine is not feasible, particularly in pediatric hematology, a field of many rare diseases. Rather, hematologists need to be able to evaluate and synthesize the literature that is availabledobservational cohort studies, administrative or claims data, or small clinical trialsdregarding a particular clinical dilemma. Several formal methods have been developed to evaluate and combine data from diverse sources addressing clinical questions that have not or cannot be answered by traditional clinical trials. Systematic reviews, such as the Cochrane reviews, use a well-defined and uniform approach to identify all relevant studies addressing a particular * Center for Innovation in Pediatric Practice, The Research Institute at Nationwide Children’s Hospital, 700 Children’s Drive, Columbus, OH 43205. E-mail address: sarah.obrien@nationwidechildrens.org 0031-3955/08/$ - see front matter Ó 2008 Elsevier Inc. All rights reserved. doi:10.1016/j.pcl.2008.01.004 pediatric.theclinics.com
288 O’BRIEN research question. The results of eligible studies are displayed and, when appropriate, summary estimates of the overall results are calculated [1]. Metaanalysis, which can be performed only if the results of studies are similar enough, refers to the statistical aspects of a systematic review, in which summary effect estimates and variance from several similar studies, statistical tests of heterogeneity, and estimates of publication bias are calculated. Formal methods of grading clinical recommendations often are used in clinical practice guidelines, taking into account the risk-benefit ratio of the treatment under consideration and the methodologic quality of the underlying evidence [2]. Decision analysis is another method of combining data from multiple sources. For many physicians, the phrase, ‘‘decision analysis,’’ may refer to an esoteric research technique involving complicated mathematics and perhaps some smoke and mirrors. In reality, clinicians use the techniques of decision analysis in almost every patient encounter. Clinical decision analysis is the application of explicit, quantitative methods to analyze decision making under conditions of uncertainty [3]. The technique originally was developed in the 1940s and used initially in financial and government sectors [4]. In decision analysis, an investigator structures a clinical problem into a decision tree representing the temporal sequence of possible clinical events. Next, data are collected to estimate the probability of each event and the expected risks and benefits of each strategy. The decision tree then is analyzed to identify which strategy has the highest expected value and, therefore, the preferred course of action. Decision analysis can be used to analyze many different types of decisions in medicine, such as determining whether or not to screen for a disease, choosing a testing strategy, or selecting an intervention. Models also can be built from many different perspectives, from individual patients or families to third-party payers to society as a whole. The possibilities for using decision analysis to address clinical dilemmas in pediatric hematology are nearly endless. Which of the following is the preferred strategy: Chronic transfusions versus hydroxyurea for prevention of stroke in sickle cell disease Partial versus complete splenectomy in patients who have hereditary spherocytosis Six weeks or 3 months of anticoagulation for a central line–related deep venous thrombosis (DVT) This review first outlines the basic steps of decision analysis: choosing a research question, building a decision tree, filling the tree with probabilities and outcome measures, and analyzing and interpreting the results. This outline is followed by a discussion of how quality of life is measured in decision analysis and the challenges of obtaining these measurements in the pediatric population. Several decision analyses published in the field of pediatric hematology and recommendations on evaluating and judging the decision analysis literature are described. Finally, the limitations of decision analysis are discussed and readers are directed to additional resources on learning about and performing decision analysis.
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PEDIATRIC ARTERIAL ISCHEMIC STROKE
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376 RODRIGUEZ & HOOTS [42] Carcao M
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378 ROBERTSON et al von Willebrand
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380 ROBERTSON et al Fig. 2. A propo
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382 ROBERTSON et al a heterogenous
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384 ROBERTSON et al of considering
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386 ROBERTSON et al Type 2M Type 2M
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388 ROBERTSON et al Desmopressin is
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390 ROBERTSON et al References [1]
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392 ROBERTSON et al [46] Nesbitt IM
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416 BLANCHETTE & BOLTON-MAGGS [16]
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422 FASANO & LUBAN of storage and t
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424 FASANO & LUBAN Leukocyte reduct
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426 FASANO & LUBAN RBCs is decrease
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Table 1 Blood component characteris
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430 FASANO & LUBAN Alloimmunization
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434 FASANO & LUBAN patients have an
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436 FASANO & LUBAN adults; therefor
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438 FASANO & LUBAN [21]. Meta-analy
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442 FASANO & LUBAN [55,56]. This is
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444 FASANO & LUBAN [20] Roseff SD,
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THALASSEMIA UPDATE 449 hypochromia
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THALASSEMIA UPDATE 451 Fig. 1. Chan
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THALASSEMIA UPDATE 453 delivery of
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THALASSEMIA UPDATE 455 thromboses i
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THALASSEMIA UPDATE 457 On the basis
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THALASSEMIA UPDATE 459 [31] Kan YW,
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ORAL IRON CHELATORS 463 large iron-
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ORAL IRON CHELATORS 465 of 50 child
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ORAL IRON CHELATORS 469 use in Nort
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ORAL IRON CHELATORS 473 in liver fi
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ORAL IRON CHELATORS 477 Other oral
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ORAL IRON CHELATORS 479 [12] Borgna
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518 TRACY & RICE [13] Bader-Meunier
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