Dear WAPO Member & Friends,
We are very thankful for the interesting presentations and your presence at the WAPO eSummit, which was held on March 19 and 20, 2021.
As promised, we’d like to share the links to the videos of the presentations and Q&A sessions. And apart from this, several presenters also answered the open questions, which were left after the Q&A session, due to the limitations in time.
Day 1 – Q&A session and Polls in English
Day 2: Q&A session and Polls in English
Q&A sessions – Open questions:
Q&A session by Johan G. Beun on ‘European Reference Centers and ENDO-ERN’
1. How do HCPs get involved in initiative in our country?
This depends on the rules in your country, in the Netherlands Health Care Professionals (HCPs) will get only the title of expert centre after ‘examination’ by a governmental committee. And the expert centres are appointed by the Ministry of Health.
2. For patients to understand all possible rare diseases is big. How do we find out specific information for specific conditions. Some symptoms are so general.
A complex and difficult question. I suggest in general to visit a large specialized hospital. Still then there is no guarantee on an (early) diagnosis.
3. How do you collaborate with the different governments? Do you advice them on new laws for example?
Mind you, the European Union started in its early stage in 1958 with six countries, now we are with over 25 countries more than sixty years later.
The ENDO-ERN thoughts, the ERN processes are a fruitful merge of the questions from the market, messages from Brussels, the opportunities of today. Apparently we are ready for it. You cannot force this if the market is still in ‘protection’ phase, i.e. look to Spain, still devided in about 15, 16 or 17 regions with all different (competing) legislations. Consequently there the success is less than i.e. in Denmark, Scandinavia, Germany and the Netherlands.
4. Has the Covid era also delayed diagnosis? Symptoms may not be addressed as assumed Covid.
Hard to say, how can you measure? What is the baseline measurement? We hear stories of delayed diagnoses in mama carcinoma, in cancer in general. So why should adrenal patients be different? I presume, I guess that pure Pituitary patients might have been able to get slightly more attention. Mind you: no science, on gut-feeling. Having said that: in the Netherlands we had in recent years ‘when live was still normal’ also every now and then people with a delayed diagnose of maybe 10 / 15 years. Thus, what is delayed and what is normal?
5. Should patients be more educated in order to recognize potential rare conditions and to push healthcare professionals investigate?
In general we advertise very much in favor of well educated citizens. In a world in which I live there is plenty of reliable Information available in our mother tongue on the web and medical specialists are easy accessible (social and in miles of distance). But if you live X-hundred kms from a hospital and medical specialists are rare with a waiting list … you are in a difficult situation. I strongly advice to all patients, patient organizations to join forces in stead of all of them making their own – incomplete – website. For instance with a thousand Euro you could translate more reliable information in Spanish or Portuguese than you make new reliable information. Thus find your best Friends amongst fellow organizations.
6. Could the ERN model be repeated in other countries or regions?
Please accept that only on good soil seed can grow. You need to have the momentum! Not all people think in harmony and cooperation. Look to what happened to the EU and the United Kingdom.
Too many people are not any more prepared to sacrifice on the short term in order to gain a lot in the rest of their lives. Most politicians are without vision and want to be reelected tomorrow. They forget in that process your and mine interest on good health on the long term.
7. Would it be possible to copy the ERN-structure outside Europe and collaborate? Like Franchise?
Very hard to say, my experiences cover only a part of this fascinating world. I have for instance never been in latin America. I think in certain countries parts of the principles, like the expertise centres, the transparency towards patients, the connection with the patient (organizations) should be able to be introduced.
It starts also with the momentum that nurses, medical doctors and patients have come to the conclusion to meet each other on the same level in the health arena to work on a cooperative model and not in a commercial service model. Look i.e. in the USA to the Kaiser Permanente model or to the Veterans Administration. Those are interesting pockets of hope in the American Healthcare System.
8. Are Pituitary adenomas counted among the rare diseases?
Yes, I think so. The most common cause of an endogenous Cushing’s syndrome is Cushing’s disease (ACTH producing pituitary adenoma), in 70% of cases.
Cortisol producing adrenal adenomas/carcinomas or ectopic ACTH-producing tumors are less common. An ectopic Cushing’s syndrome is mainly caused by neuro-endocrine tumors (bronchus, thymus, pancreas, small cell lung carcinoma, pheochromocytoma, medullary thyroid carcinoma and neuro-endocrine prostate carcinoma. Ectopic secretion of CRH occurs in <1% of cases of endogenous Cushing’s syndrome. ACTH-independent macronodular adrenal hyperplasia accounts for 1% of cases.
Cushing’s syndrome usually occurs sporadically. It can be part of a genetic syndrome (MEN1, Carney complex, FIPA, Li-Fraumeni syndrome, Beckwith-Wiedemann syndrome).
9. Are there such Centres of Excellence in Spain? This was answered by your map and also I replied on the delegate chat. If there is further information about the centres that could be supplied, that might be good.
To the best of my knowledge, there are three expertise centres in Spain:
> Hospital Sant Joan de Déu Barcelona (Barcelona)
> Hospital Universitari Vall d’Hebron (Barcelona);
> Hospital Universitario Cruces (Bilbao).
10. Please note that there are no Centres of Excellence in Latin America
I know, sorry it feels uncomfortable for me about this … but my job was to tell something about the ENDO-ERN in Europe. Yes, I feel sympathy for those that live under a different part of this planet.
Q&A session by Dr. Anton A.M. Franken on ‘Biosimilars’
1. Is there an observatory for biosimilar drugs for pituitary pathologies? In Chile we have OCTRIN, from Libra Pharma, and patients have a lot of adverse events (allergies, headache, pain, etc).
In Europe the EMA (European Medicines Agency) is responsible for registration and follow-up (adverse events ) of biological medicines in collaboration with the local agencies.
2. Among the clinical studies of biosimilars, are there new drugs for Cushing’s? Ketoconazole in tablet form is not being manufactured and in prescription form is losing quality and effectiveness.
WAPO could be a watchdog agency in this case if there is a study.
I am not aware of biosimilars in the field of Cushing disease.
3. How do agencies in each country regulate?
In Europe there is central registration for biological drugs by the EMA. Marketing authorization applications for biosimilar medicinal products, are by law reviewed centrally by the European Medicines Agency (EMA).
Based on EMA/CHMP scientific opinion, according to the requirements included in the scientific guidelines, the EC releases authorization which is valid in all (28) EU Member States.
4. Is it known how effective medicines are whose active ingredient is from India? There is a lot of myth about their quality.
If active ingredients are produced under certified GCP conditions is it safe and quality is good.
5. Where can we find clinical studies of biosimilars for acromegaly?
There are as far as I know no biosimilars underway in the field of acromegaly.
Q&A session by Prof. Javier Salvador on ‘Gonadal, Fertility and Libido’
1. How important is teamwork to manage cases of infertility associated with pituitary disorders?
Given that infertility may occur as a result of different causes the therapeutic approach usually requires the collaboration of different health care providers. When looking for advise due to infertility having a previous history of pituitary disease the first possibility that should be explored relates to hypogonadotropic hypogonadism due to hypopituitarism either from organic or functional origin. In this setting, a clinical endocrinologist with experience in assessing and treating patients with pituitary disorders represents the key component of the medical team. Regarding treatment, in some situations a neurosurgical opinion may be important if a surgical approach of a pituitary adenoma is contemplated. However, some patients may be affected by other causes of infertility caused by stress-induced psychological problems or direct gonadal dysfunction. Therefore, it is useful to have a gynecologist and, in some cases, a urologist in the medical team to rule out other etiologies of infertility in women and men respectively. The contribution of a psychologist/psychiatrist may be needed when mental disorders are suspected. Also, primary care physicians and nutritionists should also be involved to treat obesity, which is a recognized cause of anovulation and infertility. In case of gametogenesis induction is required, close monitoring by endocrinologists or gynecologists should be programmed to confirm that treatment is effective and safe.
2. Does leukoplakia indicate infection because of lower estrogen? Is it safe to take ciproflax antibiotic?
Leukoplakia is not a diagnosis, but a description of a lesion meaning “white spot”. Vulvar leukoplakia is associated with diverse atrophic and hypertrophic vulvar dystrophies. Some lesions such as the lichen sclerosus has been related to estrogen deficiency because it occurs more often in prepubertal and postmenopausal women, suggesting that low estrogen values may contribute to that problem. However, other causal factors such as autoimmune diseases, infections and even genetic factors have been mentioned as potential etiologies. Therefore, those vulvar lesions are not necessarily linked to a low estrogenic status. Personally, I do not believe that ciprofloxacin is unsafe to treat urogenital tract infections in persons with hypogonadism, but my advice is to consult a gynecologist to get an experienced opinion regarding the exact diagnosis and proposed treatment.
3. Is there any research on post (pituitary) surgery decrease in libido, when no hormonal replacement therapy is required?
As far as I know there are no solid clinical research on that topic. This is probably due to the fact that multiple factors can affect libido and to the difficulties in assessing it. If it is clear that gonadal function is maintained in the normal range according to clinical and biochemical criteria, I suggest to investigate other potential causes of libido disorders different from hypogonadism.
4. Can you get pregnant on replacement medications?
Of course, yes! Hydrocortisone or thyroxine treatment should always be maintained and adjusted as pregnancy advances, as is the case of thyroxine. Once adrenal and thyroid replacement therapy has been optimized there are no reasons to change the doses before getting pregnant. Regarding growth hormone (GH) therapy, many authors recommend to stop GH therapy once pregnancy is confirmed, whereas others prefer to continue GH administration at least during the first trimester. Desmopressin treatment should be maintained in patients with diabetes insipidus.
5. In patients with controlled acromegaly, without hypopituitarism, is it also proven that libido may be decreased?
Patients with acromegaly may develop gonadal dysfunction due to concomitant hyperprolactinemia related to a mixed GH-prolactin adenoma or to pituitary stalk compression. Hyperprolactinemia is reported in up to 25-30% of patients with acromegaly. This may apply to men and women. Further, elevated GH and IGF-1 values stimulate androgen production by adrenal glands that may affect gonadal function in women leading to reduction in libido. Mental stress and depression due to morphological changes induced by GH hypersecretion may also contribute to low libido. In general, up to 30% of patients with acromegaly may report reduction of libido.
Further, WAPO would like to thank all Sponsors – Recordati Rare Diseases, Pfizer and Ipsen, HRA Pharma Rare Diseases and StrongBridge Bio – for their support to make this eSummit 2021 possible.
The roles of Clive Deverson – moderator – and his colleagues at Cello Health Communications and designer El Cuore were very important to make the WAPO eSummit 2021 a success!
We have already started preparing the next eSummit, and will look through the topics the audience has suggested. Of course we hope to meet at our next eSummit on October 8 and 9, 2021!
The WAPO Board of Directors,
Sheila Khawaja, Muriël Marks, Sue Kozij, Sandra Mesri and JD Faccinetti