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posted by martyb on Monday April 15 2019, @10:22PM   Printer-friendly
from the YADIDNKE-Yet-Another-Disease-I-Did-Not-Know-Existed dept.

Promising Results with Givosiran in Acute Hepatic Porphyrias[*]

Investigational givosiran met the primary endpoint of reduction in the annualized rate of composite porphyria attacks versus placebo in patients with acute hepatic porphyria (AHP), researchers said here.

In the interim analysis of the phase III ENVISION trial, patients treated with givosiran experienced a mean composite annualized rate of 3.2 attacks versus 12.5 attacks in patients on placebo (P<0.0001), for a mean reduction of 74%, said Manisha Balwani, MD, of the Mount Sinai School of Medicine in New York City, and colleagues.

"We saw a robust treatment effect," said Balwani at a press conference at the European Association for the Study of the Liver (EASL) annual meeting.

"Currently, there are no approved therapies aimed at preventing the painful, often incapacitating attacks, and chronic symptoms associated with acute hepatic porphyria," she added. "The results from ENVISION are promising and demonstrate a strong treatment effect for givosiran, with reduction of attacks and improvement in patient-reported measures of overall health status and quality of life."

[*] Porphyria.

BBC has a dumbed down article: Gene-silencing: 'New class' of medicine reverses disease porphyria


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  • (Score: 0) by Anonymous Coward on Monday April 15 2019, @11:01PM

    by Anonymous Coward on Monday April 15 2019, @11:01PM (#830152)

    Your days of evil-doing are coming to an end, even if the summary doesn't bother to tell us what you are.

  • (Score: 5, Insightful) by Snotnose on Tuesday April 16 2019, @12:02AM (2 children)

    by Snotnose (1623) on Tuesday April 16 2019, @12:02AM (#830177)

    and say the dumbed down article was more appropriate. We're a bunch of computer types here, not medical types.

    I assume. I could be wrong. Then again, I don't remember the New England Journal of Medicine running articles on how to program your Linux based embedded system to implement body.arm.GetArm(BOTH).hand.fingers.GetFinger(MIDDLE).Extend();

    --
    Why shouldn't we judge a book by it's cover? It's got the author, title, and a summary of what the book's about.
    • (Score: 2) by PartTimeZombie on Tuesday April 16 2019, @12:34AM (1 child)

      by PartTimeZombie (4827) on Tuesday April 16 2019, @12:34AM (#830194)

      Investigational givosiran met the primary endpoint of reduction in the annualized rate of composite porphyria attacks versus placebo in patients with acute hepatic porphyria (AHP), researchers said here.

      The first sentence means almost nothing to me at all. I can sort of figure out what it says, except that I still don't know what porphyria is, so yes, I agree.

      Not to say that TFA is not interesting.

      • (Score: 0) by Anonymous Coward on Tuesday April 16 2019, @03:55AM

        by Anonymous Coward on Tuesday April 16 2019, @03:55AM (#830269)

        It is just jargon to make you not realize how stupid they are.

        "We gave a drug to people/rats/cells and measured a bunch of crap. A random one of the results can be spun to get desperate people to give us shittons of money if you give us shitloads of money to risk get it past the FDA."

        The FDA askes for two "statistically significant" trials. Where statistically significant is defined as p-value < 0.05. Once you go look up what a p-value means and the other data, please respond with how many you expect out of drugs that do nothing every year.

  • (Score: 0, Disagree) by Anonymous Coward on Tuesday April 16 2019, @12:05AM (11 children)

    by Anonymous Coward on Tuesday April 16 2019, @12:05AM (#830180)

    P 0.0001 means the probability of this being a fluke is less than 1 in 10,000. Very impressive.

    • (Score: 2) by Snotnose on Tuesday April 16 2019, @12:56AM (7 children)

      by Snotnose (1623) on Tuesday April 16 2019, @12:56AM (#830201)

      It's a Chi squared correlation [wikipedia.org], which has come under a bit of criticism lately. Don't care enough to google for the links, just making sure peeps know what we're talking about.

      --
      Why shouldn't we judge a book by it's cover? It's got the author, title, and a summary of what the book's about.
      • (Score: 0) by Anonymous Coward on Tuesday April 16 2019, @01:03AM (6 children)

        by Anonymous Coward on Tuesday April 16 2019, @01:03AM (#830203)

        AFAIK, the criticism applies when P less than 5 % or 1 % results are sold as statistically significant. If the number is 1 in 10,000, the result seems solid.

        • (Score: -1, Troll) by Anonymous Coward on Tuesday April 16 2019, @03:15PM (5 children)

          by Anonymous Coward on Tuesday April 16 2019, @03:15PM (#830431)

          No. If p is 1 in 10k, that means you messed up the experiment and let bias seep in (p-hacking, etc).

          • (Score: 0) by Anonymous Coward on Tuesday April 16 2019, @03:31PM (4 children)

            by Anonymous Coward on Tuesday April 16 2019, @03:31PM (#830437)

            It is a "Randomized, Double-blind, Placebo-Controlled" trial and the numbers are in TFA, so feel free to run your own analysis.

            About 50% of the treatment group had porphyria attacks, while about 80% of the placebo group had porphyria attacks. By numbers of porphyria attacks: "mean composite annualized rate of 3.2 attacks versus 12.5 attacks" in the treatment vs. placebo, respectively.

            • (Score: -1, Troll) by Anonymous Coward on Tuesday April 16 2019, @03:51PM (3 children)

              by Anonymous Coward on Tuesday April 16 2019, @03:51PM (#830442)

              While study enrollment has been completed with 94 patients in 18 countries and at 36 sites, the interim analysis

              There you go, p-hacked. They waited until the results looked good, then published something.

              • (Score: 0) by Anonymous Coward on Tuesday April 16 2019, @04:50PM (2 children)

                by Anonymous Coward on Tuesday April 16 2019, @04:50PM (#830469)

                You clearly do not know what you are talking about.

                The study is not over, but the primary outcome measure has been reported and will not change: "The annualized rate of porphyria attacks in patients with AIP. [Time Frame: at 6 months]"

                Actual Study Start Date: November 16, 2017
                Actual Primary Completion Date: January 31, 2019
                Estimated Study Completion Date: September 2021"

                https://clinicaltrials.gov/ct2/show/NCT03338816 [clinicaltrials.gov]

                • (Score: 0) by Anonymous Coward on Tuesday April 16 2019, @05:08PM (1 child)

                  by Anonymous Coward on Tuesday April 16 2019, @05:08PM (#830482)

                  Meh, don't care enough to puzzle out if this "interim analysis" was pre-planned or not. There isn't even a paper published.

                  • (Score: 0) by Anonymous Coward on Tuesday April 16 2019, @05:17PM

                    by Anonymous Coward on Tuesday April 16 2019, @05:17PM (#830485)

                    The primary outcome measures had to be declared before starting the trial. I've only heard of one or two cases where the primary outcome measures have been updated mid-trial and they need a very good reason why the new outcome measure is better.

                    Also, any analysis done before the trial is completed will be "interim" regardless of the fact that the data will not change (there will not be any additional patients or changes to the primary outcome measure).

    • (Score: 2) by darkfeline on Tuesday April 16 2019, @02:09AM (2 children)

      by darkfeline (1030) on Tuesday April 16 2019, @02:09AM (#830238) Homepage

      Wrong. P means the probability of getting this fluke given the null hypothesis is true. There's a subtle but important difference. Read it carefully.

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      • (Score: 0) by Anonymous Coward on Tuesday April 16 2019, @02:33AM (1 child)

        by Anonymous Coward on Tuesday April 16 2019, @02:33AM (#830252)

        Ahh, pedantry at its finest. But you missed the fact that "fluke" can mean: "a positive result that shows up not because of some underlying effect, but out of pure chance". In fact, that is how I meant it, and it is equivalent to your "corrected" statement.

        • (Score: 0) by Anonymous Coward on Tuesday April 16 2019, @03:18PM

          by Anonymous Coward on Tuesday April 16 2019, @03:18PM (#830432)

          There is no such thing as "pure chance". Chance is just a catchall for "no one has any idea what that happened". It plays the role of "the gods" in modern philosophy.

          There are other issues with what you said too, but I'll leave it at that.

  • (Score: 2) by Freeman on Tuesday April 16 2019, @03:23PM

    by Freeman (732) on Tuesday April 16 2019, @03:23PM (#830434) Journal

    Porphyria is a group of diseases in which substances called porphyrins build up, negatively affecting the skin or nervous system.[1] The types that affect the nervous system are also known as acute porphyria, as symptoms are rapid in onset and last a short time.[1] Symptoms of an attack include abdominal pain, chest pain, vomiting, confusion, constipation, fever, high blood pressure, and high heart rate.[1][2][4] The attacks usually last for days to weeks.[2] Complications may include paralysis, low blood sodium levels, and seizures.[4] Attacks may be triggered by alcohol, smoking, hormonal changes, fasting, stress, or certain medications.[2][4] If the skin is affected, blisters or itching may occur with sunlight exposure.[2]

    https://en.wikipedia.org/wiki/Porphyria [wikipedia.org]

    So, some sort of skin disease that can lead to death, but not contagious. That sounds like a whole lot of not fun.

    --
    Joshua 1:9 "Be strong and of a good courage; be not afraid, neither be thou dismayed: for the Lord thy God is with thee"
  • (Score: 0) by Anonymous Coward on Wednesday April 17 2019, @02:37AM

    by Anonymous Coward on Wednesday April 17 2019, @02:37AM (#830793)

    GivOS Iran: Is that a new Linux distro?

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