Some Adice From a Mom Whose Son is Battling Extensive GVHD

A. PUVA - was not helpful for us as the joints had already begun to restrict. Have David's joints been measured by PT to get a baseline? This can be an extremely helpful tool from which possible future joint involvement from gvhd can be identified. PUVA was emotionally hard. Standing naked with a homemade thong covering his private area, totally lubricated with mineral oil, eyes covered with cotton ball stuffed goggles,standing spread eagle in a "tanning booth" lead to sobbing every time. Regardless, we tried it because it would be worth it if it worked. Unfortunately it was too little too late for the gvhd had already begun to go into deeper soft tissue. PUVA doesn't go deep into skin tissue.

B. ECP - Due to David's small size he would probably have to do a style of treatment similar to ours, lots of blood products over time, massive lifestyle change and commitment,big infection risk from pheresis catheter, least toxic to organs after PUVA but this is a LIFESTYLE treatment. I would almost think of it like kidney dialysis in time commitment. Most likely would help but very open ended.

C. Sirolimus - Just tried it, noticed improvements in the skin. Unfortunately he had an adverse reaction (rare though) so he will not continue. Our dermatology doctor said Sirolimus, when tolerated well (as is usual) is much better for skin gvhd because of the T-cell impact from Sirolimus that Cyclosporin and Tacro do not have. Sirolimus must be monitored so your doctors should have some history with using this med (as with all meds) or they should be in close contact with a center and doctors who are comfortable with its use and side effects.

D. Etanercept - we are currently trying this once per week injection now. Relatively few side-effects, should be effective for skin and perhaps lung issues. Seems worth trying for the very little effort and side effects associated. I would consider this as the first option before the other choices presented.

E. I have no first knowledge about Thalidomide - sorry

Prochymal - ASK FOR THIS to be tried. Ask your doctors to contact Osiris and get a compassionate use approval if for some reason David doesn't fit the criteria. I think all our children should fit into this category but sadly our son is still not cleared for its use..... we are hanging on though. One 7yr old patient of our doctor has used prochymal. It helped initially but was not effective in stopping her gvhd. Definitely worth trying if you can light a fire under your doctors to push for its use.