A new MPN voice…The MPN-SAF-10 to Stage your MPN ..Odds of a successful SCT.
MPNforum LIVE — The living voice of the MPN Community
Jeremy Smith goes way back in the history of MPN patient advocacy. A PV paitient, he was part of the historic Harriet Gilbert group that included the late Robert Tollen and Joyce Niblack, founders of the first email patient support groups. An early member of — and columnist for —MPNforum, Smith helped build Forum over the past decade while preaching his gospel of diet and exercise for MPN patients. A strong supporter of the interferons, Jeremy started and manages the popular Facebook site MPN Life
Jeremy’s newest project, MPNforum Live, to launch later this year, will expand the international MPN global village. Just as MPNforum gave MPN patients, caregivers, healthcare providers our first full color look at each other, MPNforum Live will give the MPN community a living voice, a chance to tell our stories and hear each other’s stories.
According to Jeremy: “MPN Forum Live will interview MPN Patients, Caregivers, Alternative Medical Experts, Exercise Specialists, Nutritionists, Hematologists and others. First and foremost the platform will be focused on MPN patients and with our podcast format we will be creating a digital archive in the podcast format so that the voices and stories of MPN Patients are shared throughout our community and these voices remain with us long after we have passed on. It’s through the sharing of stories that we build stronger communities and pave a smooth road forward for the next generation of MPN Patients. The podcasts will broadcast through Apple Podcasts and Spotify.”
The elephant in the room.
MPNforum Live has a supportive but arm’s length relationship with MPNforum Magazine. Unlike the Magazine, a patient cooperative sustained by small donations, MPN Forum Live is built on a conventional and sustainable business model, staffed by employees and contractors funded through sponsorship. Approval for funding has already been received from major biopharmas whose pipeline provides the MPN patient community with billion dollar drugs.
In the past we have seen how such sponsorship has constrained other MPN patient support organizations from taking on the brutal marketing practices, clinical trial design and mismanagement, and questionable claims made by sponsoring drug companies. Aside from contractual guarantees of freedom of expression negotiated by MPN Forum Live, Smith’s commitment to MPN patients and firebrand resputation for speaking truth to power should assure objective, challenging podcasts. We’ll see….or hear.
For reactions, expressions of interest in being interviewed, program ideas and anything at all, Jeremy invites your direct contact. You can reach him at: firstname.lastname@example.org
Where does it hurt?
The MPN Symptom Assessment Form…What’s Your Stage?
When you get right down to it, myeloproliferative neoplasms (MPNs) may involve complex hematopoietic events but for patients, MPNs are about symptoms.
Symptoms are what drive patients to the doctor’s office for diagnosis. The progression or reduction of symptoms indicates whether therapy is working or not. Symptoms can affect physical abilities, emotional and psychological state, social interactions and overall quality of life.
Some symptoms are annoying, some like fatigue, bone pain, weight loss can be indicative of something more serious. The question is which MPN symptoms are most significant. And how can we correlate those symptoms with disease staging?
Until recently, even though examining physicians listened to patient reported symptoms, the real determination of MPN phenotype staget was dependent on lab results, bone marrow biopsy, and genetic testing.
Patient Reported Symptoms: The Evolution from Complaint to Diagnosis
The staging of cancer at diagnosis is critical to the planning of best treatment. Although many tests contribute to staging — x-rays, labs, biopsies, histopathology reports and other procedures, –the system most commonly used is based on tumor size, type, nature and metastatic impact.
For hematologic cancers like MPNs, there is no tumor to grade.
Based on some combination of molecular, genetic, mutational and constitutional assessments along with blood lab results, MPN disease states are usually expressed as some interval between Low, Intermediate and High Risk. The largely missing ingredient is the patient reporting how she or he feels the impact of symptoms. Since symptoms often drive patients to first MPN diagnosis and the improvemetn or worsening of such symptoms as fatigue, bone pain,, itching, bruising, weight loss etc. help determine the efficacy of treatment, symptom evaluation is an essential part of the MPN assessment and treatment processes.
And who can better report on symptoms than the patient feeling their burden?
Thinking along these lines may have triggered some of the game-changing work done originally at the Mayo Clinic/Phoenix by Dr. Ruben Mesa’s group, driven substantially by Dr. Robyn Scherber with backup from statistician AmyLou Dueck’s brilliant analysis of data, patients participating in research and testing by lots of colleagues. ( Mesa has since accepted a position as Professor of Medicine at MD Anderson, UT, San Antonio and Scherber is now runnng a research lab at Incyte Corp.)
Theor first result, the MPN System Assessment Form, incorporated patient responses to an extensive Internet survey and the Brief Fatigue Inventory developed at MD Anderson. Later refinements resulted in a shorter and clinically validated version, the MPN-SAF-10.
Drs. Mesa and Scherber’s group gave us the first element of a diagnostic scale now incorporated in MAGIC, the MPN-SAF-10. a=A precise measurement of risk for all MPN patients. MPN-SAF-10 is a scale we can all read and understand.
Survival odds after stem cell transplant.
Four years ago, when another close friend died after a delayed stem cell transplant,(SCT), a group of MPN specialists and patient advocates got together under the MPN Research Foundation umbrella. In that cloud of grief and loss, it was clear something had to be done.
SCT is the only cure for myeloproliferative neoplasm. It’s also a long and risky process. It makes sense to many enjoying a reasonable quality of life to delay the start as long as possible. It can also be a fatal delay.
Myelofibrosis patients needed precise information about their risk status to make that timing decsion.
There were good assessment scales developed over the years, clinically tested, to determine level of progression risk to blast phase or Acute myeloid leukemia. Most were unknown by patients. And hematologists unfamiliar with MPNs, as well as general physicians treating patients, needed a tool to help advise patients when to start the transplant procedure. That was the birth of MAGIC... the MF Assessment Graphic Internet Calculator.
A taskforce of 18 specialists and MPN patients worked on this tool for nearly a year, incorporating the two most popular MF assessement scales. The scales were posted ason a brief on-line quesitonnaire and the results read out with traffic light simplicity — Green, Y ellow, and Red. Those signals were accompanied by notes detailing the risk levels.
It worked. Well over a quarter million visits to the MAGIC site were recorded by myelofibrosis patients, caregivers and healthcare professionals in the past year.l.
A new home for MAGIC and the need for expanded risk assessment —
MAGIC has been run independently, until now. The MPN Research Foundation will be incorporating the tool later this year into its website.. And that means expanding MAGIC’s reach from MF to essential thrombocythemia and polycythemia vera and beyond to assessment risk after transplantation.
The scales used to determine MF risk were largely based on laboratory data. The Dr. Ruben Mesa group at the Mayo Clinic Phoenix led by Dr. Robyn Scherber has been working on a series of MPN risk assessment tools based on patient-reported symptoms. (Today both specialists have moved on — Ruben Mesa to UT San Antonio and Robyn Scherber to Incyte.)
The history of symptom stratification to determine relative severity of an MPN with clinically tested precision has a long history. An early attempt leading to our current assessment scales is the Brief Fatigue Inventory developed at MD Anderson in 1999. The Mayo Clinic expanded on this work in a pivotal 2006 publication, (The Burden of Fatigue and quality of life in myeloproliferative disorders , (MPDs) In this Mayo Clinic work, a leading MPN patient and advocate, Joyce Niblack, was a driving force.
Building on that work — greatly expanded through an on-line patient survey correlated with clinical findings — the Scottsdale Mesa Group developed and tested an 18-item MPN symptom assessement instrument against a cohort of 402 MPN patients.
Their findings:”THE MPN-SAF correlated strongly with existing clinical responses and “The MPN-SAF is a comprehensive and reliable instrument that is available in multiple languages to evaluate symptoms associated with all types of MPNs in clinical trials globally.”
With the addition of the MPN-SAF scales to MAGIC, ET and PV patients had the means to help determine current risk levels.
What was still lacking was the other end of the MPN continuum. We had the tools to pinpoint risk levels in myelofibrosis but nothing to indicate the individual patient hazard level in stem cell transplantion.. After determining myelofibrosis risk status, patients were still left with questions of their odds of a successful outcome, a cure and minimal or no Graft Versus Host Disease or long-term drug dependencies.
Enter Dr. Nicolaus Kroger, our chief transplant specialist. Kroger led a German/French group that had completed a study published in Blood outlining a scoring system to calculate the odds of a successful SCT fo MF patients.
Kroger’s developed a comprehensive clinical-molecular myelofibrosis transplant scoring system (MTSS). Variables associated with overall survival (OS) analysis were selected and then, subsequently, used to construct a multivariable mode. A full description of the MTSS, Myelofeibrosis Transplant Scoring system, can be found here
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