An opinionated take on NEJM highlights for October 2016

Continued progress in multiple myeloma

About 25,000 patients are diagnosed with multiple myeloma yearly in the US. Despite being initially treatable, typically this disease is ultimately lethal. Following a highly successful phase 1-2 study a monoclonal antibody against a marker of myeloma cells (daratumumab, Janssen) underwent phase 3 studies in combinations with established mainstays of therapy (the proteasome inhibitor bortezomib and the immune modulator lenalinomide) in a patient population several years out from their initial diagnosis.  Results were stellar, with the inclusion of daratumumab decreasing the disease progression rate by half at 1 year.  Daratumumab, Lenalidomide, and Dexamethasone for Multiple Myeloma; Daratumumab, Bortezomib, and Dexamethasone for Multiple Myeloma; Progress in Myeloma — A Monoclonal Breakthrough (subscription access)


Breast cancer: the down-side of screening exposed

A few years ago, a landmark study (also in the NEJM) showed that under reasonable assumptions regarding quality of life (e.g. what is the trade-off between lifespan and incontinence) routine PSA testing for prostate cancer was likely to have net negative utility for a male population. The reasons are that testing is imprecise, and further leads to treatment of cancers that would never have become significant within the lifetime of the patient with therapies that are not at all benign.  Now it is mammogram screening which is under the spotlight. A clever study out of Dartmouth and NCI looks at how the growth of mammogram screening has reduced the incidence of large tumors (because they are caught earlier). And indeed they have decreased, but surprisingly, the incidence of small tumors has increased much more (by a factor of 5). This can only mean that many (~80%) of small breast tumors never become large and a source of morbidity. Like for prostate cancer, it therefore seems that the hidden cost of screening is that for each detection of a small breast tumor worth treating, one detects and treats several that would never have become a threat, often with impact on a patient’s quality of life.  Breast-Cancer Tumor Size, Overdiagnosis, and Mammography Screening Effectiveness; Solving the Problem of Overdiagnosis (subscription access)


Prostate cancer: to treat or not treat is the question

Prostate cancer is very common, but is slow-progressing and a rare fraction of cases lead to death.  Initial options for management include monitoring, radiotherapy or surgery, and it’s not clear over the long run which is best. A UK-based study randomized 1600 men with localized prostate cancer between the three options and followed them for 10 years. Very few individuals died of prostate cancer (17) with a non-statistically significant excess in the monitoring group (8 vs. 5 and 4) but more people in the monitoring group developed metastatic cancer.  Those that underwent surgery or radiation had higher rates of incontinence and sexual dysfunction (and this included about 50% of the patients in the monitoring group by 10 years, presumably because their cancer progressed). Quality of life was about the same for all three groups. I can only hope that at some point, someone will do the cost analysis between the three groups (it’s NHS so someone should be able to).  10-Year Outcomes after Monitoring, Surgery, or Radiotherapy for Localized Prostate Cancer; Treatment or Monitoring for Early Prostate Cancer (free access); Patient-Reported Outcomes after Monitoring, Surgery, or Radiotherapy for Prostate Cancer (subscription access)


Bad news for the cost curve

An argument for comprehensive health insurance coverage is that it might save money reducing emergency care through better primary care.  A few years ago, Oregon conducted a natural controlled experiment of this theory through a lottery system that assigned a portion of a somewhat poor population to Medicaid coverage vs. none.  Disappointingly, the group with coverage used the ED more – some explained it away as a transient due to pent-up demand that would dissipate over time. Now in an update it is shown that this was not a transient, and that 3 years out, those with coverage have continued to use the ED more than those who did not receive coverage, thus illustrating a fundamental reality of healthcare: access begets demand. Effect of Medicaid Coverage on ED Use — Further Evidence from Oregon’s Experiment (free access)


Another sepsis drug bites the dust

Sepsis kills millions – treatment with antibiotics and vascular support often fails, therefore many drugs to mitigate the mortality of sepsis have been developed – all have failed (with the temporary exception of Xigris, but that did not end well either). Unfortunately, levosimendan is the latest to meet this fate.  Levosimendan for the Prevention of Acute Organ Dysfunction in Sepsis (subscription access)


…and finally noting the huge advertising push of Repatha (PCKS9 inhibitor from Amgen) – 6 weeks in a row on the back cover of the NEJM has to cost a lot. Wonder where the Praluent marketing dollars are going.


The New England Journal of Medicine is a premier weekly medical journal covering many topics of interest to the health sector. In this monthly series we offer an opinionated perspective on selected highlights that might be of interest to our clients and others.

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