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Position Statements

These have been approved by the BSPED and reflect the Society's views on best practice for the average patient.

Each patient must be considered as an individual in the context of their condition and other medication, and whilst every effort has been made to ensure the factual accuracy of the contents no liability can be accepted for any litigation, claims or complaints arising from the use of the statements, which is solely at the discretion of the reader.

 

BSPED and ACDC Position statement on Medtrum pump and sensor (2024)

Paediatric diabetes centres will have received the list of approved providers of HCL systems under the NHSE procurement framework. Medtrum is included on the list but centres may not be aware that at present, Medtrum has also not provided any published evidence of safety and efficacy on the use of the system in children, despite multiple requests for them to do so. The Diabetes Technology Network (DTN) have also issued a statement raising their concerns around the inclusion of the Medtrum HCL in the list of approved systems. In line with the DTN, the BSPED and ACDC (Association of Children’s Diabetes Clinicians) do not recommend that the Medtrum HCL system should be used for children and young people with diabetes in the UK until published evidence of its safety and efficacy is provided.

Dr Tabitha Randell, BSPED Chair

Dr Fiona Regan, ACDC Chair

 

BSPED and RCPCH statement on puberty blockers for central precocious puberty (2024)

The Cass Review on gender identity services for children and young people (CYP) published on 10th April 2024 has recommended that puberty blockers should only be used as part of a clinical trial in CYP with gender identity concerns. The British Society of Paediatric Endocrinology and Diabetes and the Royal College of Paediatrics and Child Health wish to make it clear that this is completely different from using puberty blockers in children who have an earlier puberty than normal. This is known as precocious puberty and defined as signs of puberty in girls before the age of eight and before the age of nine in boys. Puberty blockers in children with early puberty have been used for many decades. There is good evidence on their long term safety and efficacy, with no evidence of adverse impact on fertility, bone strength or psychological wellbeing in children who are treated for precocious puberty and thus we continue to recommend and support their use in children with precocious puberty under the supervision of a paediatric endocrinologist.

 

BSPED recommendations for the use of once-weekly long-acting growth hormone therapy in children with growth hormone deficiency (2024)

Long-acting growth hormone (LAGH) is now available in the UK and is recommended by NICE (2023) as an option for the treatment of growth hormone deficiency for children and young people aged three years and over.

Currently only one LAGH preparation is licenced (somatrogon) but other preparations are likely to become available. This guidance therefore focuses on somatrogon and will be updated when new LAGH preparations are accessible to patients in the UK.

BSPED recommendations for the use of once-weekly long-acting growth hormone (LAGH) therapy in children with growth hormone deficiency

There are separate BSPED guidance/clinical standards available for:

 

BSPED position statement ‘Growth hormone therapy in Silver-Russell syndrome (SRS)

The BSPED Growth Disorders Special Interest Group (BSPED GD-SIG) would like to draw your attention to the recently developed

BSPED position statement ‘Growth hormone therapy in Silver-Russell syndrome (SRS) between 2 and 4 years of age’.

This new position statement endorses the international consensus SRS guidelines that growth hormone treatment should be made available for SRS children from the age of two years to increase height and to optimise body composition. GH treatment should also be made available earlier if there is refractory hypoglycaemia. We recommend that integrated care boards make this treatment available in line with international guidance. 

 

BSPED statement on Glucocorticoid replacement and COVID-19 infections

Children and young people who have hormone problems and in particular who are taking steroids (hydrocortisone, prednisolone, dexamethasone) because their adrenal glands do not work properly (steroid replacement therapy) are at no more risk of catching COVID-19 than other children.

However, it is very important that all medicines are given regularly and at the doses recommended by your doctor. Make sure you have got enough medicine at home, without stockpiling, and that you follow sick day rules if you /your child becomes unwell.

Further information is available at:

NHS: https://www.nhs.uk/conditions/coronavirus-covid-19/ 

RCPCH: https://www.rcpch.ac.uk/resources/covid-19-resources-general-public 

 

CAH/Adrenal Replacement Therapy & Monitoring (reviewed in 2020)

Whilst it is recognised that multiple daily hydrocortisone dosing and 24 hr cortisol profiling may be beneficial in some patients with adrenal insufficiency, including congenital adrenal hyperplasia, whose control is challenging, there is currently no evidence to support this practice in all patients. The BSPED is currently seeking evidence to inform the development of a more detailed position statement and its members are invited to contact the Chair of the Clinical Committee.

 

BSPED Position Statement on Choice of Delivery Device for Growth Hormone Prescribing
(reviewed in 2020)

The BSPED recommends the following:

  • In accordance with NICE guidance (see below), the BSPED recommends that "the choice of product is made on an individual basis and after informed discussion between the responsible clinician and the patient and/or their carer about the advantages and disadvantages of the products available, taking into consideration therapeutic need and the likelihood of adherence to treatment."
  • The NICE guidance on choice of product should run throughout an individual’s duration of treatment with growth hormone. Treatment may require re-assessment upon transition from paediatric to adulthood services.
  • Automatic substitution (dispensing one brand instead of another equivalent or interchangeable brand) by professionals other than the prescribing team, without consultation with the hospital consultant managing the growth disorder, is not appropriate.

NICE: National Institute for Health and Care Excellence Technology appraisal guidance, 26 May 2010

Guidance 1.2

"Treatment with somatropin should always be initiated and monitored by a paediatrician with specialist expertise in managing growth hormone disorders in children. The choice of product should be made on an individual basis after informed discussion between the responsible clinician and the patient and/or their carer about the advantages and disadvantages of the products available, taking into consideration therapeutic need and the likelihood of adherence to treatment. If, after that discussion, more than one product is suitable, the least costly product should be chosen."

 

BSPED Position Statement on Paediatric Age Assessment (reviewed in 2020)

The BSPED wishes to highlight that it is not possible to accurately assess a child’s age based on physical examination or bone age assessment. Children and young people mature at very different rates and an examination can only demonstrate the stage of physical development that child is at, on that day. For example, an 11 year old girl who had an early puberty and has started her periods will be physically indistinguishable from a 15 year old girl who is at the same stage of puberty. The converse is also true – it is not possible to physically differentiate a young person who has delayed puberty from a younger child who is at the same pubertal stage. Bone age X rays will only report the degree of maturity of the bones, which is highly dependent on the child or young person’s pubertal stage and physical development. For the same reasons as given above, they cannot be used to accurately age a child or young person. For these reasons, we do not support the use of physical examination or bone age X ray assessment as tools for age assessment in children and young people.

 

Use of Prenatal Dexamethasone in Congenital Adrenal Hyperplasia (March 2023)

Prenatal dexamethasone has been available since the mid-1980s as an experimental treatment to prevent severe virilisation of a female fetus affected with congenital adrenal hyperplasia (CAH)1. Outcome studies are limited but suggest success rates of 80-85% for reducing or preventing virilisation, with low frequency of maternal complications, providing treatment is started early2. Women considering treatment need to start taking dexamethasone between 6-7 weeks of pregnancy, as it is at this time that androgens start to exert their effects and later onset of treatment may be ineffective in minimising virilisation3. With the advent of fetal sexing from free fetal DNA in maternal plasma it is possible to identify male fetuses as early as 37 days after conception, which allows treatment to be initiated only in pregnancies with female fetuses, thereby reducing the number of unnecessarily treated fetuses. A 2014 French study reported preventing virilisation in 12 girls treated before 6 weeks, and minimising changes in 3 girls treated between 6 and 7 weeks, from early diagnosis due to maternal plasma analysis.4 New et al took this a step further by analysing fetal CYP21A21 status in cell-free DNA, and targeted only affected female fetuses5. Free fetal DNA testing to establish fetal sex is now available within the United Kingdom, and non-invasive prenatal testing (NIPD) is planned.6

The beneficial effect of treatment on affected females is well established and Prader scores have been shown to have been reduced from an average of 3.73 untreated to 1.7 treated7. Concerns remain, however, about the other possible physical and psychological effects of dexamethasone not only directly on the mother, but also on the developing brain of the treated fetus. Animal studies have suggested that dexamethasone can affect areas of the brain involved in memory and programming, and can cause somatic effects including low birth weight, hypertension and metabolic abnormalities.8 However, the situation is complicated by the fact that rodents are significantly more susceptible to adverse effects of dexamethasone than primates.

There have been few robust clinical studies in humans looking at all aspects of physical and neuro cognitive outcomes on both mother and child. A meta-analysis in 20109 identified only 4 studies, which had been rigorously scrutinised, and concluded the following:

  1. Dexamethasone treatment is effective in reducing the degree of virilisation, measured by Prader score, when started early in pregnancy
  2. There was no evidence of increased risk of stillbirths, miscarriages or fetal malformations in dexamethasone-exposed pregnancies
  3. There were no differences in fetal or postnatal growth in dexamethasone-exposed or non- exposed groups but data on this were insufficient for meta-analysis.
  4. There was an increased frequency of self-reported oedema and striae in mothers exposed to dexamethasone, but no significant increase in gestational diabetes or glycosuria.
  5. Neuropsychological outcome studies suggest no evidence of reduced IQ or increased behaviour or temperamental problems, but possibly some difficulties with short-term memory and social anxiety in dexamethasone-exposed children.

Subsequent studies10 continue to express reservations and consider this treatment experimental, and in Sweden where such treatment had been offered, the programme has been discontinued due to concerns about side effects and outcomes11,12,13. However a recent meta-analysis, identifying 18 eligible studies, has suggested the outcomes are not as alarming as thought14, concluding: “prenatal dexamethasone reduced virilisation with no significant differences in newborn physical outcomes, cognitive functions, behavioural problems and temperament” but  still advising caution in interpretation, due to limitations of the studies, and recommending a “ multi-disciplinary national strategy”.

In the absence of clear unconflicting evidence or robust long-term data, and in line with the Best Practice Guidelines published by the Endocrine Society in 201815, the BSPED therefore advises:

  1. Prenatal dexamethasone treatment remains controversial and should only be considered at specialist centres that have a multidisciplinary team comprising obstetricians, fetal medicine specialists, endocrinologists, paediatric endocrinologists, paediatric urologists, psychologists and clinical geneticists. Detailed information on the current understanding and uncertainties of prenatal treatment must be provided to allow families to make a fully informed decision, and long-term follow up of all treated individuals-mothers, affected offspring and, very importantly, unaffected daughters (where targeted treatment of only affected fetuses has not been possible)- must be undertaken.
  2. All individuals who receive dexamethasone treatment in this context should be logged on to a registry (www.i-cah.org) to facilitate future data collection on the short and long term effects of prenatal treatment.

References:

  1. David H, Forest MG (1984) Prenatal treatment of congenital adrenal hyperplasia resulting from 21-hydroxylase deficiency. J Pediatr 105:799-803
  2. New MI, Carlson A, Obeid J, Marshall I, Cabrera MS, Goseca A, Lin-Su K, Putnam AS, Wei JQ, Wilson RC (2001). Prenatal diagnosis for congenital adrenal hyperplasia in 532 pregnancies. J Clin Endocrinol Metab 86(12): 5651-7
  3. Speiser PW, Laforgia N,Kato K, Pareira J, Khan R, Yang SY, Whorwood C, White PC, Elias S, Schriock E et al (1990) First trimester prenatal treatment and molecular genetic diagnosis of congenital adrenal hyperplasia (21-hydroxylase deficiency). J Clin Endocrinol Metab 70 (4): 838-48
  4. Tardy-Guidollet V Menassa R, Costa JM, David M,Bouvattier-Morel C, Houang M, Lorenzini F, Philip N, Odent S, Guichet A, Morel Y (2014) New management strategy of pregnancies at risk of Congenital Adrenal Hyperplasia using fetal sex determination in maternal serum: French cohort of 258 cases (2002-2011). J Clin Endocrinol Metab Jan 28:jc20132895 epub
  5. New MI Tong Yu K Yuen T Jiang p, Pina C, Chan A, Khattab A, Liao GJW, Yau M, Kim S-M, Chiu RWK, Sun Li, Zaidi M, Lo Dennis (2014) Noninvasive Prenatal Diagnosis of Congenital Adrenal Hyperplasia Using Cell-Free Fetal DNA in Maternal Plasma J Clin Endocrinol Metab 99 (6) : E1022-E1030
  6.  https://www.england.nhs.uk/wp-content/uploads/2018/08/rare-and-inherited-disease-eligibility-criteria-march-19.pdf [Page 291] 
  7. New MI, Abraham M, Yuen T, Lekarav O (2013). An update on Prenatal Diagnosis and Treatment of Congenital Adrenal Hyperplasia. Semin Reprod Med 30:396-399
  8. Celsi G, Kistner A, Aizman R, Eklof AC, Ceccatelli S, de Sabtiago A, Jacobsen SH (1998). Prenatal dexamethasone causes oligonephronia, sodium retention and higher blood pressure in the offspring. Pediatr Res 44:317-322
  9. Fernandez-Balsells MM, Muthusamy K, Smushkin G, Lampropulos JF, Elamin MB, Abu Elnour NO, Elamin KB, Agrwal N, Gallegos-Orozco JF, Lane MA, Erwin PJ, Montori VM Murad MH (2010). Prenatal dexamethasone use for the prevention of virilisation in pregnancies at risk for classical congenital adrenal hyperplasia because of 21-hydroxylase (CYP21A2) deficiency: a systematic review and meta-analyses. Clin Endocrinol 73:436-444
  10. McCann-Crosby B, Placencia FX, Adeyemi-Fowode O, et al. (2018) Challenges in Prenatal Treatment with Dexamethasone. Pediatr Endocrinol Rev.16 (1):186‐193.
  11. Hirvikoski T, Nordenstrom A, Wedell A, Ritzen M, Lajic S. (2012). Prenatal dexamethasone treatment of children at risk for congenital adrenal hyperplasia: the Swedish experience and standpoint. J Clin Endocrinol Metab. 97(6):1881–1883.
  12. Wallensteen L, Karlsson L, Messina V, Nordenstrom A, Lajic S (2020). Perturbed Beta-Cell Function and Lipid Profile After Early Prenatal Dexamethasone Exposure in Individuals Without CAH. J Clin Endocrinol Metab Jul 1;105(7):e2439-e2448.doi: 10.1210/clinem/dgaa280.
  13. Van't Westeinde A, Karlsson L,  Nordenström A, Padilla N,  Lajic S.(2020) First-Trimester Prenatal Dexamethasone Treatment Is Associated With Alterations in Brain Structure at Adult Age J Clin Endocrinol Metab  Aug 1;105(8):dgaa340.doi: 10.1210 /clinem/dgaa340.
  14. Xu L, Lin W, Cai L,Huang H, LiangJ, Li L, Zong L, Wang N, Wen J, Chen G (2020) Efficacy and safety of prenatal dexamethasone treatment in offspring at risk for congenital adrenal hyperplasia due to 21-hydroxylase deficiency: a systematic review and meta analysis . Clinical Endocrinology 92:109-123
  15. Speiser PW, Arlt W, Auchus J , Baskin LS, Conway  G S, Merke D P, Meyer-Bahlburg HFL , Miller WL, Murad MH , Oberfield SE , White PC.  (2018) Congenital Adrenal Hyperplasia due to Steroid 21-Hydroxylase deficiency: An Endocrine Society Clinical Practice Guideline. J Clin Endocrinol Metab 103: 4043-4088
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