4 ms·

> One of these groups (Group 1) received iloprost by vein (intravenously) for 6 hours daily for up to 8 days. The two other groups received other medications th

> One of these groups (Group 1) received iloprost by vein (intravenously) for 6 hours daily for up to 8 days. The two other groups received other medications that are unapproved for frostbite, given with iloprost (Group 2) or without iloprost (Group 3). > On day 7, the bone scan finding predictive of needing amputation was observed in 0% (0 of 16) patients receiving iloprost alone (Group 1) compared to 19% (3 of 16) patients in Group 2 and 60% (9 of 15) patients in Group 3. The presence of the bone scan abnormality was significantly lower in the two groups receiving iloprost. Most patients had follow-up information on whether they subsequently underwent at least one finger or toe amputation. The need for amputation was consistent with the bone scan findings. It seems like an effective drug based on this result.

fs_tab 3y ago
Does anyone on here know if this is due to the fact that it's a general vasodilator, or if there are specific pathways (eg: from activating prostacyclin and prostaglandin receptors) which have the effect of reducing tissue/bone damage?
sidkshatriya 3y ago
> On day 7, the bone scan finding predictive of needing amputation was observed in 0% (0 of 16) patients receiving iloprost alone (Group 1) compared to 19% (3 of 16) patients in Group 2 and 60% (9 of 15) patients in Group 3. Ah ! It would have sucked to have Frostbite not be in Group 1 and then need to undergo amputation. Talk about bad luck ! However, I totally understand that this is the main way for the science to progress and treatments to get validated.
- yjftsjthsd-h 3y ago
  The flip side is that it could have made things worse. Epistemology and ethics get complicated fast in this kind of thing:\
  - idontknowifican 3y ago
    this is why PrEP has no double blind study. it was considered unethical
- A_D_E_P_T 3y ago
  In the future this sort of thing will inevitably be viewed the way we view bloodletting with razors and leeches -- if not more harshly. They didn't know any better, way back when. We ought to know better. It's as simple as this: Double-blind, placebo-controlled studies are absolutely not required in drugs that are have extremely large effect sizes. In these cases, open-label trials, compassionate use programs, and post-marketing surveillance can be employed to continue gathering data on safety and efficacy without denying any patients access to critical treatments. And, in the end, the data you gather is just as useful, if not more useful.
  - mlyle 3y ago
    3/32 vs 9/15 is -probably- a pretty big effect size, but I am not sure how you know that a priori without trying it on a couple dozen people first and comparing. They didn’t require huge n for approval, but it took a long time to just try it on a minimal number of patients.

fs_tab 3y ago

Does anyone on here know if this is due to the fact that it's a general vasodilator, or if there are specific pathways (eg: from activating prostacyclin and prostaglandin receptors) which have the effect of reducing tissue/bone damage?

sidkshatriya 3y ago

> On day 7, the bone scan finding predictive of needing amputation was observed in 0% (0 of 16) patients receiving iloprost alone (Group 1) compared to 19% (3 of 16) patients in Group 2 and 60% (9 of 15) patients in Group 3. Ah ! It would have sucked to have Frostbite not be in Group 1 and then need to undergo amputation. Talk about bad luck ! However, I totally understand that this is the main way for the science to progress and treatments to get validated.

yjftsjthsd-h 3y ago
The flip side is that it could have made things worse. Epistemology and ethics get complicated fast in this kind of thing:\
- idontknowifican 3y ago
  this is why PrEP has no double blind study. it was considered unethical
A_D_E_P_T 3y ago
In the future this sort of thing will inevitably be viewed the way we view bloodletting with razors and leeches -- if not more harshly. They didn't know any better, way back when. We ought to know better. It's as simple as this: Double-blind, placebo-controlled studies are absolutely not required in drugs that are have extremely large effect sizes. In these cases, open-label trials, compassionate use programs, and post-marketing surveillance can be employed to continue gathering data on safety and efficacy without denying any patients access to critical treatments. And, in the end, the data you gather is just as useful, if not more useful.
- mlyle 3y ago
  3/32 vs 9/15 is -probably- a pretty big effect size, but I am not sure how you know that a priori without trying it on a couple dozen people first and comparing. They didn’t require huge n for approval, but it took a long time to just try it on a minimal number of patients.