Thursday, January 12, 2006

Therapy for IPF

I had seen this 76 y/o man about 18 months ago for "abnormal CxR". He had "dry" crackles, a mild restrictive defect (FVC, FEV1 and TLC all around 70%) and a HRCT very typical for IPF. I had discussed prognosis and treatment options and suggested a referral to the nearby University center for possible enrollment in a trial. He felt fine and declined the referral. He continued to see his PCP but had cancelled his follow-up with me. He then came back because of worsening dyspnea and hypoxemia (he is now on 3L/min O2) and his FVC is down to 45%.
I have attached a recent CT image below. He is obviously not a transplant candidate and he does not want to enroll in clinical trials. What medical therapy (if any) would you offer him?

4 comments - CLICK HERE to read & add your own!:

Arenberg said...

I hate this disease Carlos, but if the guy could tolerate it, I'd start him on azathioprine (2 mg/kg), with prednisone (20 mg/d or less). Lately, for those who are not eligible for a clinical trial, I am also starting my patients on N-acetyl cysteine based upon the recently published trial (November 24 issue of The New England Journal of Medicine). The accompanying editorial is excellent in my opinion too.

Its 600 mg t.i.d., and you ususlly just have to send the patient to the local GNC nutrition center to pick it up (Is OTC). In fact, I made the mistake of sending one patient to a pharmacy to ask for it, and they ended up sending him home with a nebulizer, and 600 mg t.i.d (inhaled!!!). Can you imagine how much nebulized mucomyst that takes? The poor guy did it for three weeks before coming to tell me the drug was making him cough. Well...no kidding?!?!?

Jeff H said...

The current "standard of care" (unfortunately) is Pred/Azathioprine + NAC. I say unfortunately, because one interpretation of the recent study (which contained NO PLACEBO ARM), is that the addition of NAC improves outcomes compared the AZA/Pred alone. The problem is that there have never been data showing that AZA/Pred is beneficial, and there are data that AZA/Pred may do harm. Additionally, the "benefit" from the addition of NAC is mild, at best.

So, the best option is to get the patient into a study somewhere. The next "best option" would be AZA/Pred/NAC if he can tolerate it. There are no data looking at NAC alone vs placebo or versus AZA/Pred.

Of course, we all know some who would argue that this patient should not be treated. This is where I'd have a very long discussion with the patient about the risks and benefits (or lack thereof) of therapy...

Baleeiro said...

I recommended trial enrollment but he was not interested. When we discussed possible therapies I tried to emphasize the lack of positive data and some of the pros- and cons-... He was started on AZA/Pred and felt better (maybe just a bit of the steroid effect). He has been on that for 3 months with stable severe restriction and I will try adding NAC on his next visit.

Eugene O said...

The NEJM study showed improvement just in physiologic parameters. Changes in dyspnea and in extent of fibrosis on CT were similar in the two groups, and no significant difference was found in mortality rates. So, no improvement in hard outcomes. I would take this as a negative study.