Friends and family expect medical professionals to know everything. For David Fajgenbaum, MD, and Denise Leonardi, MSN, both members of the Castleman Disease Collaborative Network, this started as undergrads. Sorority sisters would ask Denise medical questions because she was a nursing major. Football teammates asked David questions during medical school. As their levels of expertise grew, so did the expectation that they would knew the answers to all medical queries.
It was a rude awakening when Denise’s husband, Michael, a physician himself, became ill in 2017 with a mysterious illness initially thought to be a virus. After a month of worsening flu-like symptoms, kidney failure and liver dysfunction, the fear grew that he may have lymphoma. With multiple specialists working together, he was diagnosed after just one week in the hospital: idiopathic Multicentric Castleman Disease (iMCD).
Doctors seemed to know nothing about iMCD. Many had never even heard of it (about 6,000 individuals are diagnosed with Castleman disease annually in the US, similar to rates for ALS). According to Michael’s doctor, the good news was that it wasn’t lymphoma; the bad news was that the survival rate for iMCD was actually worse than lymphoma. Thankfully, Michael began to improve almost immediately after starting the only FDA-approved drug for iMCD and has not relapsed since.
David was similarly baffled when he became ill with iMCD during medical school years earlier in 2010. But that is where the similarities stop. David was so ill before finally being diagnosed that his last rites were read to him. He was 25. Multi-agent cytotoxic chemotherapy was the only thing that could temporarily slow down his disease, but he relapsed frequently.
So what changed between 2010 and 2017? The Castleman Disease Collaborative Network (CDCN) was founded in 2012. It launched an ambitious, patient-focused research agenda, published the first-ever iMCD diagnostic criteria just months before it was used to diagnose Michael, and recently established the first-ever iMCD treatment guidelines. Today, David is in his longest remission ever thanks to a treatment that he identified through CDCN research. David and Michael don’t know when they might relapse again, and we still don’t know that much about iMCD. But recent progress gives hope that one day we will have better treatment, and maybe even a cure, for iMCD and so many other rare and deadly diseases.
SOURCE: Castleman Disease Collaborative Network, [email protected]