Tofacitinib for Young Children with Alopecia Areata: How young is too young?

Tofacitinib for Alopecia Areata in Children

Tofacinitib (also known as Xeljanz) is an oral medication used for treatment of several inflammatory conditions. Among the best known uses is the treatment of rheumatoid arthritis.  In fact, tofacitinib is FDA approved for patients with rheumatoid arthritis that don't improve with methotrexate. 

Over the past 2 years, it's become increasingly clear that oral tofacitinib in among the most effective treatment for alopecia areata. That doesn't mean it's the first on the list to be tried - in fact, far from it.

A significant proportion of patients with advanced and treatment resistant alopecia areata will show benefit with use of tofacitinib. Side effects are not common but serious side effects can certainly occur. 2 % of patients can develop serious infections. Changes in blood counts, irritation of the liver, muscle enzyme changes, changes in heart rate, changes in cholesterol, perforation of the bowel can occur.  In some of the original trials of tofacinitib, deaths were reported.  The medication is an immunosuppressant and may increase the risk of cancer. 

I've used tofacitinib in the clinic for 2 years now and have been surprised with how generally well tolerated the medication is. There have been incredible success stories.   However, not all patients respond and I've had patients with no response or benefit at all.   I'm a weary of side effects. In fact, I'm weary of side effects with every single medication I recommend.  

Some of the toughest forms of alopecia areata to treat are those occurring in children. So the question then arises - would tofacitinib help children? There's no reason to believe it wouldn't help. Medications that help adults with alopecia areata always help children with alopecia areata.  Topical steroids, steroid injections, prednisone, methotrexate, anthralin, DPCP help adults and also help children. 

A recent study now proves that. Craiglow and colleagues from Yale published a study looking at 13 children age 12-18 with alopecia areata. 9 of them (69%) improved confirming that this drug is beneficial in children. 

The question that is raised is what about an 11 year old year old with alopecia areata? What about a 5 year old?  What about a 2 year old? I think it's clear that most would agree some cut off exists but what is it? Is it 18? Is it 12? That is were careful discussion with parents are so important.  There is no universal right answer.  There are risks and benefits to so many things and unless you have lived with alopecia areata you can not make generalizations as to what level the risk outweighs the benefits for any given patient.. 

Safety is important. We need our clinical trials to guide us with good safety data. We need government agencies to help with monitoring safety after drugs hit the market (called post marketing safety data). All of these are needed to allow clinicians to provide accurate information when patients with alopecia areata come to ask -  Am I a candidate for tofacitinib?

REFERENCE

Craiglow BG, et al. Tofacitinib for the treatment of alopecia areata and variants in adolescents.J Am Acad Dermatol. 2016 Nov 2