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Evidenced Based Management: A Journey for Physicians

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  1. Historical Perspective, Epidemiology, and Methodology
  2. Overview of the SCD guidelines and chapters
  3. Process and methodology
  4. Consensus Statements
  5. Clinical Practice Guidelines and the institute of Medicine
  6. Prevention of invasive infection
  7. Screening for Renal Disease
  8. Electrocardiogram Screening
  9. Screening for hypertension
  10. Screening for Retinopathy
  11. Screening for risk of stroke using neuroimaging
  12. Screening for Pulmonary disease
  13. Reproductive counseling
  14. Contraception
  15. Clinical Preventive services
  16. Immunizations
  17. Vaso-Occlusive Crisis
  18. Fever
  19. Acute Renal Failure
  20. Priapism
  21. Hepatobiliary Complications
  22. Acute Anemia
  23. Splenic Sequestration
  24. Acute Chest Syndrome
  25. Acute Stroke
  26. Multisystem Organ Failure
  27. Acute Ocular Conditions
  28. Chronic pain
  29. Avascular Necrosis
  30. Leg Ulcers
  31. Pulmonary Hypertension
  32. Renal Complications
  33. Stuttering/Recurrent Priapism
  34. Ophthalmologic Complications
  35. Summary of the Evidence
  36. Hydroxurea Treatment Recommendations
  37. Consensus Treatment Protocol and Technical remarks for the implementation of Hydroxyurea Therapy
  38. Indications for transfusions
  39. Recommendations for Acute and Chronic Transfusion Therapy
  40. Appropriate Management/ Monitoring
  41. Consensus Protocol for Monitoring Individuals on Chronic Transfusion Therapy
  42. Complications of Transfusions
  43. Recommendations for the Management and Prevention of Transfusion Complications
  44. New Research is Needed
  45. Data Systems That Meet the Highest Standards of Scientific Rigor Can Be Invaluable
  46. Improved Phenotyping is needed
  47. Broad collaborations for Research and Care
  48. Beyond Efficacy
  49. Look, Listen, Empathize and Ask
Lesson 48 of 49
In Progress

Beyond Efficacy

SCFA_Coach September 25, 2023

Perhaps the largest need is to translate the results of research that has been performed in laboratories and academic centers into community-based practice tools and clinical protocols. Such translational studies should lead to effectiveness trials. “Efficacy” does not always translate to “effectiveness.” One of the best examples is hydroxyurea. Although hydroxyurea has proven efficacious in RCTs, the majority of eligible persons with SCA in the United States do not yet receive this agent. Well-designed effectiveness and translational studies are needed to overcome the identified barriers that result in underutilization of hydroxyurea. These barriers include the limited number of physicians with knowledge and experience with the agent, and patients’ misconceptions and fears about side effects. Studies are also needed to examine the role of hydroxyurea in people with genotypes and clinical manifestations other than those in the MSH study. The SCD research community can make a real difference through comparative effectiveness research and other investigative strategies, as well as strengthening clinical and public health SCD collaborative efforts at the State and local levels. Finally, all of the research addressed in these guidelines cannot be successful for the approximately 70,000 to 100,000 individuals with SCD in the United States until sickle cell centers, practicing hematologists, and primary care providers in particular are fully willing and capable of taking on the challenges of serving these individuals and their families.

The expert panel realizes that these guidelines leave many uncertainties for health professionals caring for or planning to begin caring for individuals with SCD. However, we hope that these guidelines begin to facilitate improved and more accessible care for all individuals with SCD, and that the discrepancies in the data will trigger new research programs and processes that will provide the evidence necessary to expand upon evidence­-based SCD guidelines in the future.