A Closed Mouth Gathers No Feet
I have a serious medical condition and, so, I took BOATLOADS of drugs for years and also sometimes I am just not very clear-headed. ("Sometimes" she says. (snicker))
Over the years, I've gotten better at keeping my damn mouth SHUT and not saying something half-baked, half-remembered, whatever. So I like BLOGGED here the other day rather than make an ass of myself on a forum where I hang sometimes.
As noted in that post, a lot of people with CF find the FDA just CRAZY MAKING -- or, you know, DID before they came up with new drugs that kind of fix the gating issue in the cell for SOME people with CF. I left all the CF lists years ago. I don't know what they are having a cow about these days while not feeling well. (Yes, that's a thing: Sick people tend to be CRANKY. Ask me how I know.)
So my Swiss-Cheese-For-Brains memory had me recalling some discussion on some CF list about a simple sugar being denied approval due to causing lung bleeds in people with CF under age 18 and folks were up in arms about that. CF has a life expectancy of mid-thirties, so you are talking about a LOT of underaged patients and it's a big deal.
Frankly, a lot of doctors don't know what to do with adults with CF. It wasn't THAT long ago that life expectancy was EIGHTEEN.
I know because someone once posted an email to one of the lists that said "Today is my child's 18th birthday. Life expectancy for CF is currently 36. When he was born, it was EIGHTEEN."
So, anyway, people tear their hair out over what the FDA does. I didn't really pay much attention. I was busy trying to eat better, getting healthier and trying to figure out how to navigate life and ethics when I knew all these people who were DYING, medical science couldn't really fix them and everyone hated on ME for trying to share useful info, like I was trying to kill people or something.
Like, HELLO! They are already DYING. I'm supposed to stand idly by and WATCH???
Um, yeah. That's APPARENTLY what I'm SUPPOSED TO DO. Don't like care about your fellow human, no.
ANYWAY, I had seen a study years earlier into a lung treatment specifically for people with CF using xylitol and -- with the help of a friend with a medical background -- I developed a couple of xylitol saline solutions (nasal solution AND for the lungs) AND this discussion about what a meanie face the FDA was happened to be about a DIFFERENT simple sugar -- Mannitol -- that was being tested for approval with CF.
Now when I go searching the internet for "mannitol FDA," it brings up the following (edited for brevity) info:
Searching for "When was mannitol approved by the FDA" gets me the answer 1964.
I recall seeing it NOT get approved while I was on CF lists, but perhaps it was NOT approved for some specific use, I don't know. But I remember the discussion because it was part of why I made sure to put my nebulized xylitol-saline on the internet -- so MY friends with CF would have a similar treatment they could make at home and never mind what the meanie face FDA is up to. ("Meanie face" because my FOUR YEAR OLD self is being JUDGY here.)
But the internet NOW tells me this is used to treat CF when all I ever heard was it was another drug denied approval (due to it causing lung bleeds in underaged patients in trials) by the FDA that the CF community was up in arms about.
So I jotted down notes HERE in blog-land instead of opening mouth and inserting foot on HN, including this tentative "conclusion":
BUT we are IN LUCK. Someone asks:
SILVER SPRING, MD. – Uncertainty over safety and efficacy data, particularly in children, moved a Food and Drug Administration advisory panel to unanimously recommend against approval of a dry powder formulation of mannitol for treating cystic fibrosis.
At a Jan. 30 meeting, the Pulmonary-Allergy Drugs Advisory Committee voted 14 to 0 against FDA approval of the product for the management of cystic fibrosis in patients aged 6 years and older, the indication under review. The proposed dose is 400 mg twice a day, administered via a breath-actuated dry powder inhaler over about 5 minutes, using ten 40-mg capsules for the total dose.
Mannitol "hydrates the lung surface, leading to improved airway clearance," according to the Australian manufacturer, Pharmaxis. Dry powder mannitol (DPM) is approved for people aged 6 years and older in Australia and for adults in the European Union.
"I wish I could have voted yes, because I think there is a place for a drug like this, but I think that further studies are necessary," said one of the panelists, Dr. Mary Cataletto, a pediatric pulmonologist and professor of clinical pediatrics at the State University of New York at Stony Brook.
Like several other panelists, Dr. Jeffrey Wagener, professor of pediatrics at the University of Colorado, Aurora, said he may have voted in favor of approval if the indication had been limited to adults. "But there was no evidence DPM was effective in children, and "we need more information, more study, in children, particularly long term, looking at exacerbations and other outcomes besides FEV1 [forced expiratory volume in 1 second]," he added.
Pharmaxis presented the results of two phase III studies that enrolled 600 patients aged 6 years and older with cystic fibrosis (mean age about 20-23 years) with a baseline FEV1 of 30% or greater and less than 90% predicted. The trials compared treatment with 400 mg of DPM twice a day to treatment with 50 mg of DPM twice a day (controls). Patients continued other CF treatments, except for hypertonic saline, which, like mannitol, is considered an airway hydrator, and started treatment after passing a mannitol tolerance test.
In one of the studies, conducted outside the United States, the primary endpoint, the average change from baseline in FEV1 over 26 weeks, was significantly greater among those on treatment compared with controls (118 mL vs. 35 mL). But this study had a large amount of missing data because of patients dropping out, an issue raised by the FDA reviewers. In the second study, conducted in the United States, Canada, Argentina, and several European countries, the dropout rate was lower, but improvements in FEV1 from baseline at 26 weeks were only numerically, not significantly, greater among those on treatment compared with controls (107 mL vs. 52 mL).'
The safety issue that elicited the most concern among panelists and was the main focus of the safety discussion was the higher rate of hemoptysis associated with treatment, particularly among those under age 18. In the two studies, the rate of hemoptysis (reported as an adverse event, not associated with an exacerbation), was almost 11% in adults on DPM, compared with about 8% of controls. But in those aged 6-17 years, the rate was almost 8% among those on DPM, compared with almost 2% among controls, according to the company, which agreed that hemoptysis in the pediatric age group was a safety signal that needed to be evaluated further.
So I did NOT imagine it and it's why my nebulized solution is online, though these days Mannitol DOES get used for CF. ¯\_(ツ)_/¯
Over the years, I've gotten better at keeping my damn mouth SHUT and not saying something half-baked, half-remembered, whatever. So I like BLOGGED here the other day rather than make an ass of myself on a forum where I hang sometimes.
As noted in that post, a lot of people with CF find the FDA just CRAZY MAKING -- or, you know, DID before they came up with new drugs that kind of fix the gating issue in the cell for SOME people with CF. I left all the CF lists years ago. I don't know what they are having a cow about these days while not feeling well. (Yes, that's a thing: Sick people tend to be CRANKY. Ask me how I know.)
So my Swiss-Cheese-For-Brains memory had me recalling some discussion on some CF list about a simple sugar being denied approval due to causing lung bleeds in people with CF under age 18 and folks were up in arms about that. CF has a life expectancy of mid-thirties, so you are talking about a LOT of underaged patients and it's a big deal.
Frankly, a lot of doctors don't know what to do with adults with CF. It wasn't THAT long ago that life expectancy was EIGHTEEN.
I know because someone once posted an email to one of the lists that said "Today is my child's 18th birthday. Life expectancy for CF is currently 36. When he was born, it was EIGHTEEN."
So, anyway, people tear their hair out over what the FDA does. I didn't really pay much attention. I was busy trying to eat better, getting healthier and trying to figure out how to navigate life and ethics when I knew all these people who were DYING, medical science couldn't really fix them and everyone hated on ME for trying to share useful info, like I was trying to kill people or something.
Like, HELLO! They are already DYING. I'm supposed to stand idly by and WATCH???
Um, yeah. That's APPARENTLY what I'm SUPPOSED TO DO. Don't like care about your fellow human, no.
ANYWAY, I had seen a study years earlier into a lung treatment specifically for people with CF using xylitol and -- with the help of a friend with a medical background -- I developed a couple of xylitol saline solutions (nasal solution AND for the lungs) AND this discussion about what a meanie face the FDA was happened to be about a DIFFERENT simple sugar -- Mannitol -- that was being tested for approval with CF.
Now when I go searching the internet for "mannitol FDA," it brings up the following (edited for brevity) info:
- Mannitol
- Common brand names: Bronchitol
- This medication is used to treat cystic fibrosis.
Searching for "When was mannitol approved by the FDA" gets me the answer 1964.
I recall seeing it NOT get approved while I was on CF lists, but perhaps it was NOT approved for some specific use, I don't know. But I remember the discussion because it was part of why I made sure to put my nebulized xylitol-saline on the internet -- so MY friends with CF would have a similar treatment they could make at home and never mind what the meanie face FDA is up to. ("Meanie face" because my FOUR YEAR OLD self is being JUDGY here.)
But the internet NOW tells me this is used to treat CF when all I ever heard was it was another drug denied approval (due to it causing lung bleeds in underaged patients in trials) by the FDA that the CF community was up in arms about.
So I jotted down notes HERE in blog-land instead of opening mouth and inserting foot on HN, including this tentative "conclusion":
Anyway, not worth pursuing further at this time. Initial findings don't even turn up the info I recall from twenty-ish years ago and everyone already thinks I'm a flake. Bah, humbug.But then I didn't actually give up and I went and found an email. It's NOT from twenty years ago, more like TEN, but here is the title of an email from a CF list dated 1 Feb 2013:
Article - FDA panel votes against inhaled mannitol for cystic fibrosisHowever, the link listed in the email is a dead link. The article is no longer online.
BUT we are IN LUCK. Someone asks:
Can you post the text? When I hit the link, I am prompted for a user name and password.Below is what follows and it's possibly only one page of a two page article:
SILVER SPRING, MD. – Uncertainty over safety and efficacy data, particularly in children, moved a Food and Drug Administration advisory panel to unanimously recommend against approval of a dry powder formulation of mannitol for treating cystic fibrosis.
At a Jan. 30 meeting, the Pulmonary-Allergy Drugs Advisory Committee voted 14 to 0 against FDA approval of the product for the management of cystic fibrosis in patients aged 6 years and older, the indication under review. The proposed dose is 400 mg twice a day, administered via a breath-actuated dry powder inhaler over about 5 minutes, using ten 40-mg capsules for the total dose.
Mannitol "hydrates the lung surface, leading to improved airway clearance," according to the Australian manufacturer, Pharmaxis. Dry powder mannitol (DPM) is approved for people aged 6 years and older in Australia and for adults in the European Union.
"I wish I could have voted yes, because I think there is a place for a drug like this, but I think that further studies are necessary," said one of the panelists, Dr. Mary Cataletto, a pediatric pulmonologist and professor of clinical pediatrics at the State University of New York at Stony Brook.
Like several other panelists, Dr. Jeffrey Wagener, professor of pediatrics at the University of Colorado, Aurora, said he may have voted in favor of approval if the indication had been limited to adults. "But there was no evidence DPM was effective in children, and "we need more information, more study, in children, particularly long term, looking at exacerbations and other outcomes besides FEV1 [forced expiratory volume in 1 second]," he added.
Pharmaxis presented the results of two phase III studies that enrolled 600 patients aged 6 years and older with cystic fibrosis (mean age about 20-23 years) with a baseline FEV1 of 30% or greater and less than 90% predicted. The trials compared treatment with 400 mg of DPM twice a day to treatment with 50 mg of DPM twice a day (controls). Patients continued other CF treatments, except for hypertonic saline, which, like mannitol, is considered an airway hydrator, and started treatment after passing a mannitol tolerance test.
In one of the studies, conducted outside the United States, the primary endpoint, the average change from baseline in FEV1 over 26 weeks, was significantly greater among those on treatment compared with controls (118 mL vs. 35 mL). But this study had a large amount of missing data because of patients dropping out, an issue raised by the FDA reviewers. In the second study, conducted in the United States, Canada, Argentina, and several European countries, the dropout rate was lower, but improvements in FEV1 from baseline at 26 weeks were only numerically, not significantly, greater among those on treatment compared with controls (107 mL vs. 52 mL).'
The safety issue that elicited the most concern among panelists and was the main focus of the safety discussion was the higher rate of hemoptysis associated with treatment, particularly among those under age 18. In the two studies, the rate of hemoptysis (reported as an adverse event, not associated with an exacerbation), was almost 11% in adults on DPM, compared with about 8% of controls. But in those aged 6-17 years, the rate was almost 8% among those on DPM, compared with almost 2% among controls, according to the company, which agreed that hemoptysis in the pediatric age group was a safety signal that needed to be evaluated further.
So I did NOT imagine it and it's why my nebulized solution is online, though these days Mannitol DOES get used for CF. ¯\_(ツ)_/¯
