International Development Meeting – Amsterdam November 2017

Thanks to our industry partners, we traveled to Amsterdam to host a meeting with our international volunteers from Europe and surrounding countries for an International Development Meeting. We were joined by twenty liaisons for an informative day to provide updates on Research, Foundation Topics, and ways for them to host chapter meetings in their countries.

A special thank you to Global Medical Advisory Board members Dr. Bart Jacobs, Dr. Pieter van Doorn & Benson Fellow recipient Dr. Ruth Huizinga for presenting on topics such as research updates, an update on the Benson Fellowship, and on management of residuals relating to our conditions. We also thank Bruno Santoni , Executive Director, Plasma Protein Therapeutics Association of Europe for speaking with our members about PPTA and information regarding Plasma Protein Therapies & safety.

Our Foundation is so very fortunate to have a team of dedicated volunteers from all over the world. It was an honor to spend the weekend learning from one another & identifying ways we at the Foundation can better support our team, who will then spread their knowledge with their members back home!


We so very enjoyed the time we were able to spend with our extended family!

Ask the Experts

Reprinted are some of the questions from patients that we forwarded to members of our Global Medical Advisory Board. We have included the responses that we thought would be of interest to you!

QUESTION: The question is asked about life expectancy in CIDP.
REPLY: In general, life expectancy for CIDP patients is good, comparable to the general population who don’t have this disorder. CIDP can follow various courses, sometimes with recurrences or relapses over years, sometimes with a chronic progressive course. Complete remissions can occur, where the disorder seems to burn out. Rarely, respiratory failure may occur, which is relatively much more common in the acute acquired demyelinating neuropathy, Guillain-Barré syndrome (GBS), placing the patient at risk for cardiopulmonary collapse and death. Other potential causes of death in GBS, such as cardiac arrhythmias, are also quite rare in CIDP.

QUESTION: My daughter is diagnosed with CIDP and for 4 years is receiving monthly transfusions of IVIg which has limited the progression of the disease. She still suffers from pain in her hands and fatigue especially the week prior to her IVIg treatment. I have been reading about the use of Low Dose Naltrexone for patients with autoimmune disease and am interested if there is any evidence of use/success with CIDP. Any information would be appreciated. I am a donor to your organization.
REPLY: There are few if any reports on the use of naltrexone with inflammatory neuropathies such as ClDP and Guillain-Barré syndrome. Studies have been
performed using this drug in hereditary neuropathies such as Charcot-Marie-Tooth disease and metabolic neuropathies such as in diabetes and alcohol use as
well as multiple sclerosis. But it Iikely would not be easy to extrapolate from those experiences to predicting naltrexone’s potential benefit in ClDP. The patient is
described as having pain in her hands and fatigue during the week before her monthly IVIg treatment. Rather than entertaining a trial of naltrexone, it might be more fruitful to consider moving the IVIg treatments up, to every 3 weeks, to see if that approach will help relieve symptoms. For pain, such agents as pregabalin (Lyrica) and gabapentin (Neurontin) have sometimes been beneficial and are likely worth a try. The key is to start these drugs at a low dose and slowly build up the dose over weeks till there is benefit or intolerance. There is just not enough information available to offer guidance on trying naltrexone. It seems unlikely that a trial of low dose naltrexone would be harmful if its use is guided by the drug’s literature. All of these suggestions likely should be discussed with your daughter’s neurologist and/or family physician who are familiar with the particulars of her medical status and thus in an optimal situation to offer guidance.

QUESTION: I’m a physiotherapist working in rural Australia and have been asked to provide some assistance to a young boy suffering from this condition. I haven’t reviewed with him yet. But understand it took a few weeks to diagnosis and he has some postural issues, along with fatigue and lots of global weakness. If you have any protocols for physio rehab they would be greatly appreciated.
1. Ideally getting a PT and OT evaluation to work on strength,safety and activities of daily living.
2. Patients with GBS tend to have increased fatigue, for this reason the sessions should focus mainly on balance, stretching, endurance and functional adaptations. We don’t want them to get exhausted.
3. Equipment and brace evaluation is important. Patients may need AFO’s during initial recovery due to distal weakness and sometimes foot drop. We recommend the use of manual wheelchair for long distances if the patient is too fatigued, with the goal of energy conservation.
3. Ideally the session should be performed in a rehab/PT gym to take advantage of the equipment.
4. Be aware if there is any sensory loss, to educate the patient and he family about daily foot inspection and making sure the temperature of the water is appropriate.
5. Educate family about transfers and position in bed.
6. Educate the family about stretching and exercises they can do safely at home.

QUESTION: Part of my job as a Home Infusion pharmacist is initial assessments on potential IVIg patients, so I speak with many CIDP and GBS patients. I was wondering if you have any information on diet and ClDP-specifically ketogenic diets.
REPLY: That has not been formally studied for ClDP that we know of. Some recommend an anti-inflammatory diet, which is avoiding foods high in saturated fats. Eat more fruits and vegetables. Foods thought to reduce inflammation include olive oil, dark leafy vegetables like kale, spinach and broccoli, oily fish like salmon, tuna, mackerel, and sardines. Almonds, ginger root, low-fat yogurt, tomatoes, beets, sweet potatoes. This again has not really been studied but is a reasonable diet anyway.

QUESTION: I have a damaged peroneus and I can’t push my feet up, on the hands I do not have fine motor skills. They told me that this damage is from a vaccine named POLIO. That happened to me when I was 3 years old, now I’m 20. I’m interested if there’s a solution for this and can
you help me? Can I renew my nerves?
REPLY: Damage to peroneal nerves and loss of fine motor skills can be due to several disorders. It is unlikely that these current problems reflect the effects of a vaccine received so many years ago. Rather, it would likely be prudent to consider other causes, such as hereditary (e.g., Charcot-Marie-Tooth disease) or acquired peripheral neuropathies. More information than is provided in the query would likely be helpful to hone in on the underlying diagnosis. It will likely be best to obtain a hands-on evaluation by a neurologist who specializes in neuromuscular disorders and/or peripheral neuropathies. Your local medical school’s university teaching faculty likely has such specialists in their neurology department. The neurologists of the Center of Excellence of the GBS|CIDP Foundation are also suggested as a reliable resource to obtain an evaluation. Best of luck. A timely evaluation is encouraged since earlier treatment of some disorders
may lead to less risk of more nerve damage.

QUESTION: My question for medical expert is: I only receive 55 grams IVIg (has been every 3 weeks since end of April, will be every 2 weeks after this plasma exchange). The other man I know about (different doctor, my age-74, who weighs 1 1/2 times what I do) has been getting treatment (after loading), 5 days straight x 3 times, now 2 days x 2 times, way more grams, is improving happily, was first seen by hematologist to target his IVIg. I have no health issues in my life (till the back surgery last November which threw me into CIDP) except right breast cancer mastectomy 11 years ago, and I do take RX for high blood pressure and for hypothyroid. How many more grams may I take?
REPLY: Different CIDP specialists have different approaches to how they dose IVIg. Having said that, the most common strategy is to give 2 grams for every kg
of body weight as an initial dose and then give 1 gram of IVIg per kg every 3 or 4 weeks. The number of days of treatment is not the important factor; the total amount of IVIg given is the important factor. A person’s age and other medical issues determine how quickly IVIg can be given. A relatively young and healthy person can get 1 gram per kg of IVIg in a single day. In an older patient we may divide that dose over 2 or 3 days (some physicians are cautious and give IVIg over several days in everyone). Some CIDP specialists give less than 1 gram per kg per month. Surprisingly, there is not much research to determine the optimal IVIg dose. However, if someone is not doing well it makes sense to give a higher dose. The bottom line is that it would be reasonable for you to receive at least 1 gram per kg of IVIg every 3 weeks. Some physicians will give as much as 2 grams per kg of IVIg every 3 weeks. That would generally be the maximum.
Hope this answers your question satisfactorily. (to get your weight in kg divide your weight in pounds by 2.2).

QUESTION: Is there a standard protocol that they follow to detect any cardio respiratory regression?
REPLY: For GBS in the acute setting spirometry should be measured frequently, between twice daily and 4 times daily depending on the clinical concern for respiratory compromise. When expiratory FVC decreases below 15cc/kg/body weight or NIF declines to less than 20 cm H20, urgent intubation and mechanical ventilation should be strongly considered. If these respiratory parameters are precipitously declining but have not reached these thresholds intubation should also be performed on a semi-elective basis to avoid urgent intubation at untimely hours or uncontrolled settings.

QUESTION: How hard should one push the patient (especially patients who are say, gym instructors and already attuned to muscle fatigue and/or a certain amount of pain-tolerance)…are there any guidelines in this respect that cater for specific patients like these? That is, if physiotherapy pushes the selected few GBS patients, who ultimately want to be pushed, in a gym style workout rotating limbs, etc. are we causing deleterious effects?
REPLY: There are no formal guidelines to counsel patients on activity following GBS but we know that ability appropriate escalation of exercise is good. It has
been shown that post-GBS patients have the capacity to improve fitness and that exercise improves fatigue (a common residual in GBS). Caution is advised with isometric exercises in muscles that are weak, as this may induce deleterious damage to muscles that are already impaired. I discourage patients from exercising weak muscles until failure. I encourage regular exercise and activity, even in weak muscles, that is appropriate to the patients ability level, safe, focuses on flexibility, and is aerobic.

2017 PNS Meeting GBS|CIDP Research Projects

The following research projects relating to GBS|CIDP were presented at the 2017 Peripheral Nerve Society Meeting in Sitges, Spain.

Filip Eftimov, MD, PhD
Department of Neurology, Academic Medical Center, Amsterdam, Netherlands.

Chronic Inflammatory Demyelinating Polyradiculoneuropathy (ClDP) is a remarkably heterogeneous disorder with various clinical presentations. Despite different sets of diagnostic criteria , not all patients with treatable ClDP are identified. Several challenges remain during treatment, such as which treatment should be started first, when are patients adequately  treated and when can treatment be stopped. In other words, there is an unmet need to improve diagnostic criteria and find clinical or biological variables that can predict treatment response, disease activity and long-term outcome. To address
these questions, it is required to conduct a long-term prospective study with a large group of ClDP patients collecting
highly standardized data and biomaterials.
In recent years, several national registries and biobanks have been developed. However, even in large countries, these registries will not be able to include sufficient patients to address the most important challenges described above. Recently a multidisciplinary group of 24 people from 13 countries attended the European Neuromuscular Center (ENMC) workshop. Eight currently ongoing international ClDP registries that included a total of over 1300 patients were compared to assess infrastructure and collected clinical data, diagnostic data and biomaterials. During this workshop a consensus was reached on several important issues to make comparison between these registries possible. A central database (INCbase) will be used to upload data from current registries and databases while these registries continue to exist.
Next steps are to harmonize the current registries protocols and to set up INCbase. This global database is expected to be operational in 2018 and will collect data from thousands of ClDP patients to enable solving some of the important challenges in diagnosing and treatment of ClDP.

Towards an affordable treatment of GBS for patients from low-income countries
Badrul Islam, MD, PhD Fellow
icddr,b, Dhaka, Bangladesh, Erasmus MC, Rotterdam, The Netherlands

Guillain-Barré syndrome (GBS) takes its toll on the resource poor developing countries where the incidence of GBS is several fold higher than that of Europe and North America. In Bangladesh, 15% of patients with GBS die and 20% remain unable to walk. The poor outcome of GBS in these countries is explained predominantly by the lack of treatment and medical support. Specific treatment for GBS with either intravenous immunoglobulin (IVIG) or plasma exchange (PE) in Bangladesh can be afforded by a small minority of patients and more than 90% of patients receives only supportive care.
Our aim was to develop a safe and effective treatment for patients who cannot afford the expensive IVlg or PE. We developed a new technique that is based on the same principle as PE, removal of neurotoxic antibodies and other components from the blood, but is 25 times less expensive. In this Small Volume Plasma Exchange (SVPE) blood is obtained from patients and cleared from the plasma just by gravity. This procedure can be done by the bedside of the patient, without the use of electricity and complex machinery. We started with a pilot study to define the feasibility and safety of the procedure using a strict protocol in 20 patients with GBS in the National Institute of Neurosciences and Hospital in Dhaka, Bangladesh. This study showed that the procedure indeed is feasible and safe during a follow-up of 6 months. We are very thankful to the GBSICIDP Foundation International for funding part of this study. Based on this study we cannot conclude on the efficacy of the treatment that needs to be defined in a new study in a larger number of patients. To conduct this efficacy study, further funding is required. If the efficacy is demonstrated, SVPE may become available to treat GBS in patients from low-income countries that are currently untreated. About more than half of all patients with GBS in the world belong to this category.

Marielle Pruooers
Universitv Medical Center Maastrict, The Netherlands
IgM paraprotein associated polyneuropathy is a slowly progressive disease with numbness or tingling of arms and legs and severe imbalance. Occasionally, tremors and weakness are present. These symptoms are disabling, but difficult to measure. In addition, the disease is relatively rare, not very well-known, and there is no cure available. The IMAGiNe study aims to gather more knowledge about the disease by creating an international database of a large group of patients. From these patients we will learn more about the history of the disease, neurological and hematological disease characteristics, and the response to current or past treatments. The IMAGiNe study will also evaluate current outcome measures and develop new outcome measures, hereby focussing on patients’ needs. These outcome measures will represent the impairments, disabilities, quality of life and treatment expectations. The study has started in the Netherlands in 2016, and currently 90 patients are included. In 2017 the study will also be performed in the United Kingdom, Spain, Serbia, Italy, France, Switzerland, and the United States of America, aiming to include at least 400 patients worldwide. The knowledge gained from the IMAGiNe study will serve as a starting point for new therapeutic trials in the near future.

A new treatment with compliment inhibition improves motor function in Guillain-Barré Syndrome: Japanese eculizumab trial for Guillain-Barré Syndrome (JETGBS)
Satoshi Kuwabara
Department of Neurology, Chiba University Hospital, Chiba, Japan
Recent basic studies on the pathophysiology of Guillain-Barré syndrome (GBS) have shown that complement activation has a major role on peripheral nerve damage in GBS. Eculizumab (Alexion Pharmaceuticals Inc) is a humanized monoclonal antibody against complement C5, and is approved to treat complement-mediated blood diseases. At the Peripheral Nerve Society Congress (8-12 July 2017, Sitges, Spain), Japanese GBS study group presented results of a randomized placebo-controlled phase 2 trial with eculizumab in patients with severe GBS (unable to walk). At 24 weeks, marked improvement in motor function was observed in patients treated with eculizumab; 74% of patients in the eculizumab group (n=23) regained ability to run , compared with 15% in the placebo group (N=11).

A dose response trial of IV immunoglobulin in Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)/DRIP study
Professor K. Kuitwaard
Erasmus MC, University Medical Center, Rotterdam, The Netherlands
In clinical practice ClDP patients are treated with several dosages of intravenous immunoglobulin (IVlg) as well as with different intervals. It has never been investigated what the optimum dosage and frequency of IVlg in ClDP is. The effective component of IVlg is IgG. More frequent dosing will lead to more stable IgG levels in the blood as well as higher trough levels (the level directly before the next dose) which may lead to an improvement in muscle strength. Furthermore, more frequent dosing will lead to lower peak levels (the highest level in the blood) which may lead to less side effects.
It has never been investigated whether high frequency low dosage IVlg treatment is more effective than low frequency high dosage. In the DRIP study we investigate if:
1. A more frequent infusion at a lower dosage is more effective in ClDP.
2. A more frequent infusion at a lower dosage leads to less side-effects.
3. A more frequent infusion at a lower dosage leads to more stable blood levels.
It has been shown that a lot of ClDP patients are still receiving immunoglobulins even though they do not need treatment any longer. Therefore we will check before the start of the study whether patients still need IVlg treatment.

From the Executive Director

Dear Friends,
As always at year’s end, we tend to reflect on the year past. So much has happened this year that recapping would need its own newsletter!

On the following pages, you will read of some of our accomplishments, events, research, activities, etc. None of these could have happened without all of you! Of
course, our Board of Directors, Global Medical Advisory Board, Chapter Liaisons, Walk-n-Roll chairs, and Points of Contact are all identified and we are grateful for the amazing job they do!

But it is the micro-volunteers who also deserve acknowledgement, those of you who are an unnamed part of our family, who are out there also
carrying out our missions of support, education, research, and advocacy. For example: patients who request extra materials to give to their doctors; young people requesting bracelets for a “pop-up” school activity; off-time volunteers to exhibit at their local health fair.

Thank you to those who contribute to our social media pages. We can go on and on. What a great organization! What a great family! What greater message can I send to you but the best of health, happiness, and peace for the New Year!

Lisa Butler
Executive Director