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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 44 of 49
In Progress

New Research is Needed

SCFA_Coach September 25, 2023

Cure is always the most desirable outcome for any chronic disease. Therefore, research that increases the evidence for and availability of a cure for SCD is a high priority. Hematopoietic stem cell transplantation (HSCT, formerly called bone marrow transplantation) is a treatment option for an increasing but still small number of people with SCD. The procedure involves “conditioning” therapy, utilizing myelosuppressive and/or immune-modifying drugs, followed by infusion of histocompatible stem cells (derived from bone marrow, peripheral blood, or umbilical cord blood). Substantial risks are involved with the procedure, and it is not yet feasible in the majority of people with SCD. Although clinical trials have provided promising results, and cure appears to be possible in a large proportion of patients receiving HSCT, additional research is still needed that addresses the potential risks of this therapy (e.g., failure of engraftment and chronic graft-versus-host disease) before HSCT can become a widely used therapy.

Additional research is also required to address the many other areas with little or no evidence that were identified during the development and writing of these guidelines. The needed studies include observational work to better describe the utility of screening asymptomatic individuals with SCD for commonly occurring chronic diseases; studies to better describe the clinical course of the occurrence and treatment results of all the acute and chronic complications of SCD; comparative effectiveness studies to provide clear outcomes on best approaches to SCD and its complications;clinical trials for new therapeutic approaches or to improve on current therapeutic approaches such as examining the role of hydroxyurea in people with genotypes and clinical manifestations other than those in the MSH study or transfusion goals in chronic conditions. A few of the other larger research agenda issues, in addition to the need for an SCD cure, are summarized below.


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