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The “rare patient”

The “rare patient”. Julia del Amo ISCIII, Madrid, Spain. Rare patients from cohort studies can inform public health policy . The Translational Power of Collaboration: Harnessing Large Datasets to Understand the HIV/AIDS Epidemic and to Optimize Care and Treatment.

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The “rare patient”

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  1. The “rare patient” Julia del Amo ISCIII, Madrid, Spain

  2. Rare patients from cohort studies can inform public health policy • The Translational Power of Collaboration: Harnessing Large Datasets to Understand the HIV/AIDS Epidemic and to Optimize Care and Treatment

  3. Definition of “rare” from the Oxford dictionary • (of an event, situation, or condition) not occurring very often • (of a thing) not found in large numbers and so of interest or value • unusually good or remarkable

  4. – common • – frequent • – abundant • – ordinary Synonyms and antonyms of rare • Uncommon • infrequent • Scarce • extraordinary

  5. To find the rare ones within large numbers of common events • To contextualize them to value their “uniqueness” To find uncommon events you need:

  6. What is the role of Extremely Large Datasets in HIV/AIDS and Global Cohort Collaborations regarding “rare patients” ?

  7. International cohort collaborations allow identifying meaningful numbers of "rare subjects" What is the role of Extremely Large Datasets in HIV/AIDS and Global Cohort Collaborations regarding “rare patients” ?

  8. Who are rare subjects in HIV/AIDS ? Rare “Exposures” and “Outcomes” Outcomes Exposures Those with uncommon and/or remarkable features

  9. Clinical presentations • Response to treatment • Socio-demographic characteristics • Disease progression patterns • Biological traits

  10. “Rare exposures” • Socio-demographic characteristics • Women in Western epidemics • Children in Western epidemics • Migrants from specific origins • Distinct ethnic minorities • People over 70 years

  11. “Rare exposures” • Socio-demographic characteristics • Women in Western epidemics • Children in Western epidemics • Migrants from specific origins • Distinct ethnic minorities • People over 70 years Depends on the setting

  12. “Rare events”

  13. Long-term non-progressors and elite controlers • Rapid and very rapid disease progression • Viraemic non progressor • Seroconverters • with narrow windows - primoinfected • During pregnancy • HIV-2

  14. Understanding Pathogenesis • Long-term non-progressors and elite controlers • Rapid and very rapid disease progression • Viraemic non progressor • Seroconverters • with narrow windows - primoinfected • During pregnancy • HIV-2

  15. Vertical transmission despite PMTCT • Treatment in infants • Presenters with < 50 cells/mm3 • ART response in non-B HIV-1 subtypes • Uncommon adverse effects of ART • Cause-specific mortality in specific groups • Women • Migrants • Ethnic minorities

  16. Policy Implications • Vertical transmission despite PMTCT • Treatment in infants • Presenters with < 50 cells/mm3 • ART response in non-B HIV-1 subtypes • Uncommon adverse effects of ART • Cause-specific mortality in specific groups • Women • Migrants • Ethnic minorities

  17. What is rare in a setting may be common in another • Except for some groups such as migrants from Sub-Saharan Africa, the proportion of women in the HIV epidemic in Europe has been < 25% • In Sub-Saharan African cohorts, women account for > 50% of the subjects

  18. Men Women Deaths 803 430 Deaths without AIDS 272 130

  19. WE NAME SSA LA Asia Deaths 2195 13 26 25 10

  20. Three axes- gender, ethnicity and social class – conform social health inequalities

  21. Three axes- gender, ethnicity and social class – conform social health inequalities • Gender, ethnicity and social class should be viewed as EFFECT MODIFIERS rather than confounders requiring stratified analyses

  22. Three axes- gender, ethnicity and social class – conform social health inequalities • Gender, ethnicity and social class should be viewed as EFFECT MODIFIERS rather than confounders requiring stratified analyses Incorrect assumption that unmeasured confounders are equally distributed within these three axes

  23. Study key outcomes by geographical origin and sex in COHERE: • Appropriateness of the timing of cART initiation and treatment interruptions • cART Virological and immunological responses • All-cause and cause-specific mortality

  24. High numbers High power Low data quality Misclassification Non-differential

  25. High numbers High power Low data quality Misclassification Non-differential

  26. Strategic questions on rare subjects need to be formulated within large datasets

  27. Strategic questions on rare subjects need to be formulated within large datasets • quality control procedures • using standardized definitions • to provide the adequate statistical power for its translation into public health policy

  28. Founding networks

  29. Founding networks Thank you for your attention

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