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Acute promyelocytic leukemia can be treated without chemotherapy

MD-FM Thursday December 20, 2012

MD FM — Medical News from around the world with Peter Goodwin.


And with me is Sarah Maxwell. To begin with, patients with acute pro-myelocytic leukemia can be treated without chemotherapy, according to findings announced last week at the American Society of Hematology annual meeting in Atlanta…

Yes, a randomized phase three trial looking at patients with newly diagnosed, non-high-risk, acute pro-myelocytic leukemia showed: outcomes with a combination of all-trans retinoic acid — or ATRA — and arsenic trioxide, were at least “not inferior” to ATRA in combination with idarubicin chemotherapy...

Hmm, of course ATRA was the big step forwards in acute pro myelocytic leukemia because this reforms the cancer cells instead of exceuting them, but then chemotherapy was deal with cells that didn’t respond and the combination has been the gold standard for newly diagnosed patients…

Yes and and you get roughly 80% cure rates, but toxicity with chemo has always been the problem, triggering: infections, nausea, hair loss and so on, and according to Professor Francesco Lo-Coco and his team in Italy arsenic trioxide can be just as effective as chemo with fewer side effects:
Bob-Lo-Coco: “Recently, arsenic trioxide, which was used as a remedy in many diseases was shown to be very effective specifically in acute promyelocytic leukemia, and carried much less toxicity compared to chemo. Because these studies were small studies with limited number of patients, we reasoned: why don’t we try to compare the two approaches: the standard chemo + retinoic acid vs. the arsenic + retinoic acid, and this is what we did in this study. And to our surprise, we did see similar or even better outcomes with significantly less toxicity for patients with no chemo.”

That was Francesco Lo-Coco, from the University of Tor Vergata in Rome.


Also at the American Society of Hematology meeting: for patients with resistant and refractory multiple myeloma, who’ve exhausted most standard treatment options, a new immunomodulatory drug: pomalidomide in combination with low-dose dexamethasone was being offered as a potentially a new standard of care...

Yes, as with previous imids, pomalidomide was given in combination with either low or high-dose dexamethasone. In Atlanta, Meletios Dimopoulos told us about his group’s results from a randomized trial looking at 455 patients with resistant and refractory multiple myeloma:

bob-Dimopoulos: “The main outcome of the study is a significant improvement in the progression free survival in favor of pomalidomide and low dose dexamethasone, which was about 4 months, versus 2 months for high-dose dexamethasone. A secondary endpoint was also met, which was a higher response rate in favor of pomalidomide/low-dose dexamethasone, which is right now 16%, but we expect this to increase over the several few months, and also there is a survival advantage in favor of pomalidomide/low-dose dexamethasone”
Professor Meletios Dimopoulos from the Alexandra Hospital in Athens, Greece. And he also said there were some expected toxicities in both groups, including neutropenia, thrombocytopenia and fever, but overall the combination was well tolerated.



And also, briefly, from American Society of Hematology meeting: for people with relapsed or refractory multiple myeloma, high doses of an anti-CD38 inhibitor called daratumumab — a single-agent monoclonal antibody and the first targeted therapy for these patients — reduced bone marrow plasma cells and para-protein levels, in a phase I/II study. There weren’t any big safety issues with daratumumab: adverse events were: brief low-grade infusion related reactions; and it’s the first time results like this have been demonstrated with a single-agent monoclonal antibody.



For people who’ve suffered a whiplash injury, treatment which is more intensive — and more costly — does NOT speed up recovery compared to basic care, according to findings published in The Lancet…

Yes, emergency department staff were given enhanced training — in a large randomized study from the UK — to provide “active management” consultations. This included: six-physiotherapy-sessions for patients with persisting symptoms. But it wasn’t any more effective than the usual consultations and only had modest benefits compared to single-physiotherapy-advice sessions. Lead study author Sarah Lamb:

Bob-Lamb: “Treatment effect was really pretty small, and by the time you looked at the cost of delivering that treatment in comparison to... you’ve got standard benchmarks looking at the cost-effectiveness of treatments.. it just didn’t stack up in terms of cost. So emergency departments should continue to do what they do at the moment, and if patients have persisting symptoms, they should just have one additional session of physiotherapy. So the implication is that health services would save money… I think it’s going to be very interesting to see what the insurance companies make of this –I  really don’t know what their take will be on it, but it has quite profound implications for the amount of money that is spent both in the NHS on these type of injuries and more broadly through the motor insurance industry.”

That was professor Sarah Lamb, from the University of Warwick in the UK.




For patients with the most severe subtype of juvenile idiopathic arthritis — systemic JIA — a randomized study looking at the first drug ever approved for treatment has shown encouraging results…

Yes, safety and efficacy data on 112 patients randomly given placebo or the interleukin-6 inhibitor — tocilizumab — were published this week in the New England Journal of Medicine:

Bob-De-Benedetti-1: “One year after beginning tocilizumab, 50% of patients are in what we define inactive disease state –no clinical signs of arthritis, normal labs, no symptoms, no signs of disease –which is a very surprising result for this disease. And more than 50% of the patients were able to withdraw from treatment with glucocorticoids. So this is extremely important, especially considering the severity of the population that was enrolled in this study.”

That was study author Dr Fabrizio De Benedetti, from Ospedale Pediatrico Bambino Gesù in Rome. He added that side effects linked to tocilizumab are common — albeit expected — and there’s a long-term safety surveillance study ongoing.

In the same issue of the New England Journal, another study showed — canakinumab, an interleukin-1 inhibitor — was also effective in systemic JIA. Dr De Benedetti again:

Bob-Bedenedetti-2: “We now have 2 targets in a disease in which we had none. We have one drug which is approved, we have one drug which is anakinra, which can be used off label and a third drug, which will soon become available... so we have a therapeutic armamentarium that will probably change the history of the most severe form of arthritis in children.”

Results from both these trials will shed light on the mechanisms behind this difficult-to-treat disorder and these two agents signal a new era in the treatment of systemic JIA.


Finally, in brief:
Chronic alcohol and marijuana use during youth can compromise white-matter integrity, according to a new study: Alcoholism: Clinical & Experimental Research that confirms previous findings. Longitudinal changes in fiber tract integrity, associated with either “excessive” or “minimal” substance use, were analyzed using diffusion tensor imaging in 92 young people over 18 months.


Scientists have developed a mind-controlled, robotic, prosthetic hand with an unprecedented degree of control and freedom of movement! Two micro-electrode arrays were implanted into the left motor cortex of a person who is tetra-plegic. By just the second day of training, the patient was able to move the hand without the help of a computer — a huge step forward in the development of robotic prosthetics. The details are online first in The Lancet.

That's all from MDFM for now. We’ll be back next week with the first of two Christmas special editions, so until then from me, Peter Goodwin, goodbye!


Previous editions


Best of Science in Nutrition 2013: Yogurt for a healthier diet (EB & IUNS 2013)

Best of Science in Nutrition 2013: Yogurt for a healthier diet (EB & IUNS 2013)
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