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Making Sense of What we Read about Scleroderma Treatments

Making Sense of What we Read about Scleroderma Treatments. Kimberly Watkinson September 19, 2014. Objectives. E valuate medical information found on the internet. Understand some concepts of evidence-based medicine.

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Making Sense of What we Read about Scleroderma Treatments

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  1. Making Sense of What we Read about Scleroderma Treatments Kimberly Watkinson September 19, 2014

  2. Objectives • Evaluate medical information found on the internet. • Understand some concepts of evidence-based medicine. • Be familiar with evidence behind scleroderma treatments, including stem cell transplantation. • Able to use knowledge to assist with making informed decisions.

  3. Evaluating Medical Information on the Internet • Who runs the Web site? • What is the purpose of the site? • What is the original source of the info? • How is the info on the site documented? • How is info reviewed before it is posted? • Good sources: sites end in .gov; .edu; .org

  4. Be wary of terminology such as “innovation”, “quick cure”, “miracle cure”, “exclusive product”, “new discovery”, “magical discovery”, “secret formula”, “suppressed by Government”

  5. Evidence Based Medicine (EBM) What is EBM: • A decision-making framework that facilitates complex decisions. • Considers research and evidence. Concepts: • 1. Study design • 2. Sources of bias • 3. Sample size • 4. Measures of precision

  6. 1. Study design • Descriptive • Case study • Observational • Case-control (retrospective) • Follow- up (cohort, longitudinal, prospective) • Cross-sectional • Experimental • comparison groups, investigator • **Randomized Controlled Trials (RCT)**

  7. 2. Sources of Bias • How was the study population selected? • How were patients allocated to groups? • Were the groups observed differently?

  8. Controlling for bias • Randomization • Blinding • Single blind: when either the patient or investigator does not know • Double blind: neither the investigator nor patient knows which group patient was allocated to.

  9. 3. Sample size(number of patients in study) • There is uncertainty introduced by studying a “sample” of the population (random error). • The larger the sample size, the more confident that the benefit of a treatment found in the study represents the true effect.

  10. 4. Measures of precision P-values (P= ____) • The smaller the P value, the stronger the evidence against the result being a fluke. • P < 0.05 is considered “statistically significant”.

  11. “Proven Therapies” for scleroderma • Interstitial Lung Disease (ILD) • Cyclophosphamide • Skin • Methotrexate • Cyclophosphamide

  12. Scleroderma Lung Study II • 2- year course of mycophenolatemofetil compared to • 1- year course of oral cyclophosphamide

  13. Autologous Bone Marrow Transplantation Rationale: • Intense immune suppression followed by an immune reset • Alter inflammation and autoimmune component

  14. Autologous Stem Cell transplant Stem Cell Collection Thaw & reinfusion cryopreservation High Dose therapy Patient Mobilize stem cells Stem cell collection High Dose therapy Give back stem cells

  15. Evidence 3 randomized clinical trials: • ASSIST • Single center study • Non-myeloablative conditioning regimen • Results: all ten patients allocated to transplant improved (P= 0.00001) • ASTIS • Trial ongoing • Multi-center study in Europe • Non-myeloablative conditioning regimen • SCOT • Trial ongoing • Multi-center study in North America • Myeloablative conditioning regimen (includes TBI)

  16. SummaryStem cell transplantation • Most effective therapy shown to reverse skin fibrosis. • Awaiting results of ongoing trials. • Appears to change the natural course of scleroderma. • Not a cure. • Role in therapy likely for select patients: • early diffuse systemic sclerosis at risk of early mortality • exclusion of high-risk candidates.

  17. Interpreting info • Is there scientific evidence (not just personal stories) to back up the statements? **** However promising experiments in animals or anecdotal clinical experience, or how widespread, such observations can not predict the results of appropriately designed RCT. ****

  18. Antibiotic Protocol (AP) • Based on theory that disease caused by mycoplasma infections. • Many anecdotal reports of success What is the evidence?

  19. Antibiotic Protocol (AP) • Open-label trial • n= 9 • Results at 1 year (Total skin score): • 4 pts had complete resolution of their skin disease • 2 patients no improvement • 1 patient improvement

  20. Health Professional perspective • Evidence-Based Medicine background • Role to recommend therapies with well-established efficacy

  21. Patient perspective • Don’t care about evidence • Importance of hope • Sense of control over life • Personal preference

  22. Reality • A lot of research needed • Limited evidence for current therapies • Huge variability in disease course between patients • Right disease to be open minded

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