ASH Myeloma news: is a complete response really a Complete Response?

Multiple myeloma is a rare and dangerous bone and blood malignancy affecting thousands of Americans each year. The disease had minimal effective therapy until relatively recently with the advent of Thalomid its derivative drug Revlimid and the intravenous Velcade. These three medicines have altered the landscape of myeloma treatment. Myeloma is said to be "multiple" because of the bone fractures it can cause in multiple locations. Diagnosis is usually made on the basis of a bone marrow aspirate and blood tests. Prior to the clinical availability of the three effective drugs above research focused on stem cell transplantation as a strategy to treat myeloma. In this procedure the patient's own blood stem cells are harvested stored while the patient gets very high-dose chemotherapy then reinfused as a rescue agent. The procedure is expensive and inconvenient but has gotten safer in recent years. Multiple research studies have shown a long-term cure rate of 20-30% and some centers are performing multiple transplants to enhance the rate of cure. A controversy has arisen recently in hematology over whether transplant or chemo are superior in the treatment of myeloma. There are many specialists who argue that the results with the new medical therapy are so good transplant is not necessary any more. The results of therapy are good transplanters say but since they've only been out a few years nobody really knows what the long-term efficacy will really be. This year the debate raged on as researchers presented data on combined treatment with Revlimid and Velcade (therapy expected to cost $100 000+ per year). The following is a reprint of a report from the conference about the combined therapy http://www.tmcnet.com/usubmit/2008/12/15/3857782.htm Outstanding response rates were reported. According to a hematologist who attended ASH there was considerable debate during the presentation. The argument centered on the significance of a complete response. Generally "responses " or signs of improvement of a disease are easy to come by in hematologic malignancy. A much more elusive goal remains avoidance of relapse. A mentor of mine once tried to persuade me not to go into heme-onc by telling me that in the field your successes go away while your failures surround you. Nowhere is this more true than in malignant heme where relapse can play out in the hospital over a peri0d of years. It sounds to me like even some of the non-transplanters are starting to ask whether response rate is a valid measure of efficacy. Different fields have different definitions of efficacy. Pancreatic cancer researchers use softer endpoints like quality of life because survival improvements are so hard to demonstrate in that field. Colon cancer researchers are still debating whether progression-free survival is equivalent to survival and I can't tell you how many papers have been published on this very boring subject. What I do is consider transplant for every patient with myeloma. You get different chances to talk about it with them and there are a lot of educational resources out there for them. http://myeloma.org/main.jsp would be a good start. Some people would say "No way not for me." I have a 90-year old lady for example getting low-dose Velcade for her hip pain and she is not a transplant candidate. Some people are interested in transplant. I don't think you can risk-stratify and only send the high-risk people or low-risk people or whoever for transplant eval. I think there isn't any one group that transplant benefits most. Would I personally want to do it? I would probably want to get a good remission with Velcade or Thalomid or Revlimid then consider definitive transplant at some point. The meds we have are expensive and have side effects and I'm not sure most would want to be on them for many years. Better to seek a treatment with a chance for cure a concept all too rare in our field.