Among the 688 myeloma-related abstracts presented at the Annual Meeting of the American Society of Hematology (ASH) this year, one stands out. This is “Late-Breaking Abstract: LBA-1,” presented by Dr. Ed Stadtmauer on Tuesday, December 6th at 7:30 am. The StaMINA trial enrolled 758 patients and was a randomized study comparing single ASCT (autologous stem-cell transplant); single ASCT plus 4 cycles VRd (Velcade®, Revlimid®, dexamethasone) consolidation; and double ASCT; all with lenalidomide (Revlimid) maintenance. The analysis conducted with 38 months of follow-up showed no differences between the three arms: A NEGATIVE study! Thus, I would say, much to everyone’s surprise, neither the second transplant nor 4 cycles of consolidation added benefit.

Probing questions

Could this be true? Immediately, myeloma experts at the ASH meeting asked probing questions. Was the assessment—at around three years—too soon? Would differences emerge later? Dr. Stadtmauer responded with an emphatic: No! Statisticians evaluating the data felt secure that major differences were unlikely to emerge later. Well, how about high-risk patients? Prior and other data presented at this year’s ASH meeting suggested benefit for high-risk patients. Again, the answer was no, but it was less emphatic because high-risk information will not be available for all patients and a definitive answer may not be possible. How about a subset of patients with deeper responses, maybe even MRD negative? Again, unlikely to be a major subgroup.

One transplant is enough

As the questions subsided, the reality set in that patients do not need to have a second transplant nor consolidation. This is great news! The intensive additional therapies had always been a burden. Now, with relief, both doctor and patient can know that one ASCT, plus lenalidomide maintenance, is enough. The toxicities, the time, the costs, the family disruption can all be set aside. Truly good news!

Clarity and simplicity

Add to this the clear recommendation that a triple-therapy frontline treatment, such as VRd (or “VRd lite”) can be strongly recommended across the board, and one has a very simple treatment plan (or “algorithm”) to offer for newly diagnosed myeloma patients. This clarity and simplicity are enormously welcome.

Extraordinary outlook heading into 2017

More welcome news from ASH are the tremendous successes of the daratumumab combination therapies (DRD [POLLUX data: (Abstract #489 and Abstract #1151)] and DVD [CASTOR data: (Abstract #1150 and Abstract #3313)]). These findings mean we have decisive positive recommendations for management of early relapse. Given expectations for initial and first-relapse therapies, the average outlook for a newly diagnosed myeloma patient is 7 to 10 years heading into 2017. Really extraordinary, when one thinks about what further new therapies can likely emerge by 2025!

Skyrocketing cost of melphalan

Just as every cloud can have a silver lining, bright clouds of hope can also have elements of caution and concern. A single transplant using high-dose melphalan is definitely recommended for eligible patients. However, melphalan costs have skyrocketed! The Italian government recently sued the manufacturer (South African-based Aspen Pharmacare, who recently bought a portfolio of GlaxoSmithKline drugs including melphalan) for illegally raising the price by 1,500% (from a few hundred to a few thousand dollars)! The French government has refused to pay. A tragedy ensued when three French lymphoma patients died after treatment in which cyclophosphamide was substituted for melphalan. The melphalan was being saved for myeloma patients, considered to be a higher priority for melphalan use. This is the beginning of a complicated story about costs and access to melphalan. We will keep you updated as new information emerges.

For now, the silver lining is that myeloma patients have a clear, simpler, less toxic path to best outcomes. Good news indeed!

Image of Dr. Brian G.M. DurieDr. Brian G.M. Durie serves as Chairman of the International Myeloma Foundation and serves on its Scientific Advisory Board. Additionally, he is Chairman of the IMF's International Myeloma Working Group, a consortium of nearly 200 myeloma experts from around the world. Dr. Durie also leads the IMF’s Black Swan Research Initiative®.

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