The Year Ahead for MHAUS-2009

As the New Year begins, it is customary to look ahead and plan and predict what is likely to occur in the coming 12 months. It’s always dangerous to engage in such predictions, but nevertheless, here are my thoughts.

The basis for my outlook is the published literature related to MH and allied disorders, the clinical information about MH that we learn from the MH hotline calls, the discussions among the hotline consultants, abstracts presented at various meetings and conversations and comments from our professional advisory council members and MH experts in other countries. In addition we have been listening to you. We have been analyzing our calls, e-mails, inquiries and conversations at exhibits and patient conferences in order to develop programs and materials better suited to meet your needs—from patients to basic scientists, from students to experienced health care professionals.  On the basis of these inputs, here are some of the things that I think will occur in 2009.

Consensus Guidelines:

Transfer of Care Guidelines

As I have written previously, this effort is ongoing to develop guidelines for the safe transfer of patients who develop MH in a non-hospital setting (e.g. ambulatory surgery center) to a hospital for treatment of an MH crisis.  This effort began following the tragic deaths of several patients who developed MH in a non-hospital setting and succumbed after transfer to the hospital. It is being supported by joint funding from MHAUS and the Ambulatory Surgery Center Foundation.  This important project is an interdisciplinary effort, including anesthesiologists, nurse anesthetists, emergency medicine physicians, emergency medical transport personnel and those who work in ambulatory surgery centers. This guideline will inform providers as how best to manage an MH episode in the critical time period during transfer of care. As far as I know there is no similar consensus guideline in existence.

Interestingly, a report in this month’s journal Anesthesiology based on national hospital data shows that the mortality from MH when a patient is transferred into a hospital from another setting is several fold higher (20%) than when the episode begins in a hospital (Rosero, EB et al. Trends and outcomes of Malignant Hyperthermia in the US, 2000 to 2005, Anesthesiology, January 2009, page 89; full text available on line after February 2009 at www.anesthesiology.org).  I will have more to say on this issue next month.

Consensus Guideline for the Diagnosis and Treatment of NMS

We have also just received funding from Astra Zeneca to develop a consensus guideline for diagnosis and treatment of Neuroleptic Malignant Syndrome (NMS). NMS, which resembles MH when it occurs in a severe form, occurs rarely in patients who receive drugs that block dopamine activity in the brain. We plan to fulfill an important need for standardization of diagnostic criteria and a protocol for treatment based on available scientific evidence.

Testing for MH

The introduction of a DNA test for MH susceptibility in 2002 has been a great advance. However, the majority of DNA changes (variants) that have been found in MH susceptibles have not been characterized as to whether they really lead to the changes that cause MH (i.e., causative mutations). This year will see a greater understanding of the role of those DNA changes in MH. The short-term goal is a test that will detect over 80% of MH susceptibles in an accurate, inexpensive manner. The long-term goal is even greater accuracy. At present if a causative mutation is found in an individual, family members who have the same mutation are clearly at risk. With greater accuracy and understanding it is hoped to be able to tell those family members who do not share the mutation that they are not susceptible. We cannot do that at present.

In addition, there is evidence that DNA changes in genes other than the ryanodine receptor gene will be identified and thus clarify why some MH susceptibles do not show any DNA changes in the ryanodine receptor gene.

We have recently posted a slide presentation that reviews the options for diagnostic testing for MH susceptibility including muscle biopsy and genetic testing on the MHAUS web site.  While this information is geared toward medical professionals, we hope that MH susceptible individuals and their family members will find the content valuable, especially as an aid for discussions with their doctors.

Information Concerning MH and NMS That is Easier to Understand

Although we make every attempt to explain all aspects of MH and NMS in a manner that does not require a science or medical background, we hear from many that we need to do a better job.For example, some of you want to know what choices the anesthesia provider has in delivering anesthesia care and why one is chosen for a particular procedure and patient.  Also, what are the anesthesia providers actually doing when they care for the patient under anesthesia? What are the monitors and why isn’t temperature monitored in all patients? Why are MH trigger agents still in use?  It is true that major anesthesia societies have addressed these and other issues in a manner that the general public can understand, the least we can do at MHAUS is provide links to such material or, with their approval, post them on our web site.   In addition, when we review our printed material we will also try to carefully explain all terminology and complex issues.

MHAUS Website

Speaking of our web site, www.MHAUS.org currently receives over 2 million visits/year. Our information must, therefore, continue to be updated and of high quality.  All the material posted on the website is carefully reviewed by members of our hotline and professional advisory council.  The same is true for material posted on the website for NMS and serotonin syndrome (NMSIS.org). Hence, our information is reliable and up-to-date. MHAUS has become the “go to” site for reliable information on MH.

Dantrolene

As you know there are now two companies, JHP Pharmaceuticals and US World Meds who sell dantrolene in the US. JHP purchased the original product from Procter and Gamble, while US World Meds markets a generic version of the drug.  Both are investigating methods to promote more rapid solubility of the drug, so that valuable time will not be spent in mixing the drug as is the case at present. Yet another more soluble formulation of dantrolene (Ryanodex) has been studied in animals by another company. However, as far as I know the formulation has not been reviewed by the FDA.

Programs for Training in the Management of MH

We also have learned that many nurses who care for patients in the OR, recovery room, and same day surgery facilities want to learn more about MH. For example, most of the attendees at our patient/professional conference in September 2008 were nurses.

Furthermore some of the ambulatory center accrediting organizations require an annual MH drill. Many centers already do this. We will work on developing a “kit” or guidebook on how to perform such a drill in a meaningful manner in different environments.

Another way that health care professionals learn how to deal with complex medical situations is through the use of simulation. Simulation takes many forms, including a”dummy” or manikin that can be programmed to display signs of MH. But there are other, low tech simulations.  For example, at next year’s American Society of Anesthesiologists meeting, we will have a workshop on what it means to be an MH Hotline Consultant. This will draw on cases from the MH hotline. Volunteers will play the role of the hotline consultant with an actual hotline consultant being the “caller”.

Progressing at a slower rate, because it is fairly complicated, is the development of a program using the manikin simulator to enable anesthesia providers, surgeons and nurses to recognize, treat and mange an MH crisis in a realistic environment. Again, certain simulation centers do this already; however, it is important that the simulations are accurate and reflect many different presentations of MH and syndromes that may resemble MH.

These are just some of the highlights for the coming year. Here are others that are on the table:

1.       A webinar related to MH

2.       A patient/professional conference

3.       A consensus statement and monograph on MH in the ambulatory setting

4.       A conference for hotline and professional advisory council members to keep up with the rapidly changing developments in the field

5.       Better understanding of the risks to the MH patient from environmental situations and other drugs

6.       Further studies of MH using the data stored in our North American MH Registry

7.       Expansion of the use of our Facebook ”cause “ page

8.       Translation of MH material into other languages

9.       Development of subsidiary websites geared specifically to the needs of specific countries

10.   Information on how to create and retain a family health history.

11.   Creation of a repository for tissue samples taken at the time of muscle biopsy diagnostic testing for studies on the molecular genetics of MH.

All of these ventures and programs take place against the background of the usual, ongoing activities of answering hundreds of calls/month; supporting the MH and NMS hotlines; publishing our E-newsletter and the Communicator; exhibiting at meetings; maintaining our web site; etc.

We therefore have a full and ambitious agenda for the year. We are, however, fortunate to have wonderful commercial, individual, and professional society supporters. We are also fortunate to have over 50 professionals who donate their time and expertise to MHAUS and NMSIS activities.

It promises to be an exciting year with major developments in the understanding of MH, and in the recognition and management of MH and NMS. All with the aim of eliminating death and disability from MH, NMS, serotonin syndrome and related disorders.

As always, your comments are appreciated.

Have a Happy and Healthy New Year.