Science & Medicine

The Patient Playbook

by Zhenya Senyak

It’s hard to be an MPN patient.

On one hand, the MPN world is on a roll.  Money is pouring into research, new more powerful and better targeted drugs are already in clinical trial.  One or more cocktails of drugs seem likely to halt MPN progression and roll back the worst of symptoms.

On the other hand there is persistent difference of opinion by experienced MPN hematologists over treatment options and diagnosis.   And then there’s the problem of affordable access to qualified hematologists and varying insurance company policies on drug approvals.

Compounding our difficulties as patients and caregivers are our own illusions.

While the Medical-Drug Complex parades its accomplishments, hand in glove with universities, cancer centers, hematologists and clinical investigators, the patient stands on the sidelines hoping for some benefit.  Expecting, or at least hoping, to be cared for.

That’s not the way the system works.

Last week, The New York Times reported on the premature baby scandal  a government-financed studyNEJM Baies with participation of major universities like Stanford, Yale and Duke. Researchers, according to the Government report,“… had sufficient available information to know, before conducting the study, that participation might lead to differences in whether an infant survived, or developed blindness, in comparison to what might have happened to a child had that child not been enrolled in the study.”  Parents were not told.  You can read the study results in the chilling New England Journal of Medicine report and never suspect behind all the high class science-babble that babies were blinded or died because of the study protocols.

Sometimes, that’s the way the system works.

We have to be alert, understand our options, and forcefully advocate for ourselves…or enroll someone who can. 

-> Two weeks ago, when one of us progressed from MF to AML  he was promptly dropped from one of the new JAK2 inhibitor trials. He now has no meds, no hematologist, no therapeutic path forward.

-> Last week, many of us read on Facebook of a severely anemic MPN patient denied Procrit  by her insurance company. She was grateful to be approved for a costly blood transfusion and its risks only after her physician intervened on her behalf.

-> Also posted on Facebook, a patient without insurance called a major medical center for a consultation with an MPN specialist.  She was told it required a $5,000 deposit.

That’s the way the system works.

 In a world where professionals are playing gritty hardball, why are we so meek in our pursuit of health services?


Our doctors, the good ones anyhow, well understand how little is known for certain about myeloproliferative neoplasms. Among hematologists, there are strongly held, often loud and opposing convictions, about almost everything relating to MPNs:   What it is…what causes it…how to treat it…everything.

Added to the legitimate scientific and academic uncertainty, we are in an arena where hard science often takes a back seat to high finance, where dollars and euros drive the flow of drugs into the pipeline and finance fuels the line of patients queuing up for clinical trials.

As patients we tend to passively accept the judgment of men — and more rarely, women —  in white coats and antiseptic medical settings It’s a costly attitude that often leads to painful outcomes.  Our misconceptions and real need for relief and hope can blind us to the realities facing us as patients with a rare and deadly disease.

 There are some good solid options for us but there are few bona fide magicians out there. And we can get hurt choosing the wrong doctor, the wrong treatment.

At a minimum, we need to understand the shadowy system we have fallen into.  The Patient Playbook is one look at some elements of that system and our menu of options.

The menu

There are strong advocates for  hydroxyurea …and there are those who consider its long-term use a recipe for acute leukemia.  Some opinions are well-informed, experience-based, some views come from patients or doctors with a bias. Is it a good, well-established safe drug that’s going to keep us from stroking out or is it cytotoxic and slowly poisoning us? Where’s the science?

There are those who swear to the efficacy of Pegasys and those who think the interferons might have a place in therapy but are no miracle drug and far from risk free.

The thalidomide derivatives, the HDAC inhibitors, the ruxolitinib-type JAK2 inhibitors RX warning fda drugin their various flavors….what can we reach for with any certainty in a world where an eminent professor of hematology can loudly urge us to enter a clinical trial rather than take an FDA-approved drug?

And then there’s stem cell transplant, considered the only “cure” for MPNs.   To succeed, stem cell transplant requires an experienced transplant team, a highly compatible donor and an MPN patient strong enough to accept the rigorous conditioning and recovery period. And even then it comes with a price.

SCT is not available for the great majority of MPN patients who, generally, don’t acquire this blood cancer — if it even is a blood cancer — until later in life.  Old or young, by the time the SCT decision needs to be made, too many of us are already too weakened by our MPN or another unrelated condition to qualify.

None of our choices are easy…or certain.

Our last choice may be the most attractive, the most risky and the most contaminated:  The Clinical Trial.

It’s most attractive since some of the new drugs being rolled out for clinical test, aimed at new targets, do indeed have extraordinary promise.  Some may well be the ultimate drug or combination that rolls back fibrosis and stops our MPN cold.

It’s contaminated because MPN clinical trials are funded at a mega-million dollar rate, far out of proportion to our numbers. This tsunami of cash pays salaries for physicians, nurses, techs, clerks, and janitors, pays the facility’s light bills and supplies and is the main driver of MPN medical research in America. (The reason for all this attention is we have had the luxury, for the past six years, of having the JAK-STAT pathway implicated in our MPNs, a bond with more common and  profitable diseases.) Equally good or better options don’t even get to the preliminary study phase since the financing is not available.

And the risks are real. The first studies of a drug in humans, the dosage and toxicity studies, are undertaken when there is some indication of worthwhile activity.  Patients are given escalating doses of a drug and closely monitored. We are taken off drug or given reduced dosage when exhibiting Adverse Effects. Severe Adverse Effects can be deadly and weakening.  Those who worsen significantly drop out of trial or are removed by the clinical investigator.

These Phase I/II trials are risky but there are benefits, of course:  A high level of “free” medical care and often the promise of a lifetime supply if the drug is successful. And if not successful?  Well, it’s a clinical trial isn’t it.

The gold standard, Phase III clinical trials, double blind studies measuring the MPN drug against a placebo or a known other drug, carries fewer risks since the dosage and toxicity issues have been somewhat sorted out.  But while we call it a trial or a study, this is still an experiment, an experiment on human subjects with an unknown outcome. For us, the carrot is a cure…or at least symptomatic relief.  For the sponsors, the carrot is profit, and for many clinical investigators the reward is enhanced reputation, advancement, greater opportunity to secure new grants.   Some are in it to advance research, some specialize in clinical trials as the best place for them to help relieve patient suffering.

We don’t have to sort out motivations and rewards…but it helps to be aware of the process before we sign on.

The results of the clinical trial are reported in learned journals, at ASH and other meetings.  These reports include graphically supported statistical tables often prepared with the aid of sponsoring medical facilities and Big Pharma marketing departments.

We need to study those data and charts since, because for patients, they make up what is essentially the Odds Board. These colorful charts usually stress the extent to which a particular drug was proven effective in specific circumstances. It becomes the basis, once the drug is approved for sale, for us to place our bets through our doctors.  Or have our doctors place our bets.

Les jeux sont faits. The bets are down.

It’s a high-stakes bet played out in our bone marrow and large organs.

Usually, we lose. The House always wins… but luck and skill can improve our odds..

Nobody’s evil here, the system is just rigged, shot through with money and competition for resources. With luck and skill, we can make it work for us.

The two players we most rely on – hematologists and drug companies – have their own realities. And our medical insurers theirs. (Curiously, the same profit driven machinery that has provided almost all the medical and technical breakthroughs we depend on is responsible — through our bizarre medical insurance system —  for making those same breakthroughs unaffordable or difficult to access for many patients.)

Hematologists are generally not primary care physicians, not medical missionaries or Doctors without Borders.  They’re a tight fraternity of highly paid specialists. Almost all are smart, most seem honestly to care about their patients and some are greedy.  They had to complete a long and costly education and apprenticeship, pass tough boards and accept a period  of deferred compensation to qualify. We need a primary care physician but even more we need a dependable  hematologist on our team to improve our odds.  Finding one is not easy.

In our search for a hematologist, a working partner,  fame and reputation is no guarantee of successful treatment and might actually be an obstacle.  Sometimes the better choice is the full-time clinician who  is not distracted by writing papers for publication,  traveling and making presentations, balancing the demands of a study with the demands of rounds.  The clinical investigator is a specialist among specialists, deeply knowledgeable about a subset of potential remedies, closer to the clinical implications of specific drugs.  On the other hand, it’s not surprising to run across a hematologist who has never seen a myeloproliferative neoplasm patient.  We need referrals, recommendations. We need to talk to each other about our experience of physicians.

Using the MPNforum List of  patient-recommended hematologists actively just makes sense. Many patients who listed their physicians are also willing to talk about their experience.  The search for a good hematologist partner is hard work.  It may take awhile.

The other player we rely on, Big Pharma, is simple to understand Pharmaceutical company execs serve at the pleasure of a Board of Directors for the benefit of the corporation’s stockholders. They are in business to make a profit.  The good ones do good work. And sometimes that work trickles down to us at the dripping end of the drug pipeline.  As patients, we don’t have to pay much attention to the individual corporations involved beyond recalling how much power they wield in the vast system that brings drugs to us.

As MPN patients our most serious hurdle is internal: Attitude. 

Instead of pooling our experience with other MPN patients, too often we withdraw.  Instead of pressing our doctors for details and, when necessary, clear translation of terms, we nod and take notes. And, worst of all, instead of studying our options, we take the most convenient path.

We have the option of tossing in our cards or passing now and then instead of just betting the hand that’s dealt us.

Take me back to the Contents

© Zhenya Senyak and, 2013. Unauthorized use and/or duplication of this material without express and written permission is strictly prohibited. Excerpts and links may be used, provided that full and clear credit is given to Zhenya Senyak and with appropriate and specific direction to the original content.



Comments on: "The Patient Playbook" (1)

  1. Kathy Van Meter said:

    It’s all true, Zen. Good job. And even having an acknowledged expert in the field does not mean treatment will be easy. We are all at the mercy of front and back office staff who stand behind statements like, “unless it’s a true emergency we have 48 hrs. to return your call.” Or, “I sent the paperwork to your insurance company, don’t know what happened from there.”
    And when you righteously contact the medical facility’s Patient Advocate you run the risk, often realized, of setting up an adversarial relationship with the doc’s gatekeepers. Those of us with ANY chronic disease requiring treatment must be mighty warriors, keen researchers, diplomatic communicators, all the while dealing with the good, the bad and the ugly of our daily lives. Peace……………….Kathy

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