Cure JM Foundation 2018 National Family Conference Recap

General Session Q&A

Blood Draw for Genetic TestingOne of the most popular parts of the General Session features a panel of world-renowned JM researchers that take questions from the audience. This year, we ran short on time, so we collected the remaining questions, and connected with the members of our Medical Advisory Board to answer those questions. Susan Kim, M.D., MMSc, Lauren M. Pachman, M.D., Ann M. Reed, M.D. and Lisa G. Rider, M.D. graciously took the time to reply.

Should you, or shouldn't you, take folic acid the same day you give an MTX shot?

Dr. Rider: It is theoretically better not to take folic acid on the same day as the MTX shot. This is because folic acid works in the metabolic pathway of methotrexate and would help it metabolize more quickly. So, when methotrexate is on board, one would effectively be receiving a lower dose.

What is the standard practice you often use to manage post IVIG side-effects?

Dr. Rider: There are several things to manage side effects: use Tylenol with Benadryl before and during the infusion. Use a pulse dose of steroid before the infusion. Slow the rate of the infusion so it goes in over more than 8 hours. Pre-hydrate prior to the infusion - with oral hydration and/or IV fluids (a bolus of normal saline). Another very effective approach is to switch brands of IVIG to a preparation that has lower sugar load and lower viscosity in the IVIG preparation.

Are children ever treated with just IVIG & no steroids- has that been tried?

Dr. Reed: Yes, there have been a few case series where IVIG alone has been used primarily in adults with less than good response when used in isolation. I also have known patients who have been treated with IVIG alone who have come to me for an opinion have needed additional therapy.

If aldolase & VWF fluctuate but CPK is normal consistently does that child still qualify for medicines?

Dr. Reed: All of these measures can be varied between children and also can normalize even if you have active disease. Specifically, CPK is more likely to normalize the longer you have disease and VWF is abnormal when you have blood vessel involvement so not all children have abnormal VWF. So, these labs in isolation cannot determine disease activity but instead it is a combination of labs, history and physical exam.

Have you ever used plasma exchanges (3 times a week) and also Baricitinib as therapies in case of refractory JDM? Our doctors in France told us it will be the next step in case of new relapse. What do you think/know about efficiency?

Dr. Kim: In general, plasma exchange is reserved for very sick patients, since it involves large, repeated infusions of blood products, typically requires special access, and can be associated with some serious complications. There is some report of benefit for some patients. Patients with myositis antibodies may theoretically benefit more from plasma exchange. We are excited about the possible benefit of medications like baricitinib/JAK kinase inhibitors in Juvenile dermatomyositis- and there is a compassionate program for this at the NIH: that is in process. More studies are needed for JDM. It is a reasonable medicine to try if other conventional medicines have not helped your child's condition.

Would it make sense to perhaps combine B cell targeted drug (Rituxan) and T cell targeted drug (Orencia) or would that be too aggressive a "hit" on the immune system?

Dr. Reed: For patients with severe disease, or those resistant to the best treatment protocols that have been proposed, you do consider combination of medications including Rituxan with other medications. What we don't know is what are the possible complications of adding biological agents together compared with the benefit. It does make biological sense to combine some of these agents however risk of side effects, especially infections, are theoretically much higher.

Please discuss "combo" treatments? How aggressive (3+drugs) is too aggressive?

Dr. Pachman: [This question] is driven by the type of JDM illness that the child has and their response to previous therapies.

If trying to taper/reduce IVIG, is it better to decrease dosage or frequency?

Dr. Kim: This question was also asked during the Cure JM meeting - and there were different approaches to tapering IVIG: some of our experts decreased the dose of IVIG rather than the frequency. In my experience, most patients do well with slowly decreasing the frequency of IVIG - I try to do this in most cases, in hopes of limiting time spent out of school/missed work and activities, etc.

Additional note from parents at the conference: If the patient has a port-a-cath, please discuss these options with the patient's physician. In some cases, decreasing the frequency of IVIG infusions may require the addition of an additional appointment to "flush" the patient's port-a-cath between infusions.

Video: Panel Question and Answer