Abstract
Objective
Adolescents and young adults (AYA) with common endocrine disorders show a high dropout (up to 50%) after the transfer from paediatric to adult endocrinology. Little is known about transition readiness in rare endocrine conditions (rEC). This study aims to assess medical self-management skills (SMS) among AYA with rEC in relation to age and gender, in order to understand dropout and increase transition readiness.
Design
Cross-sectional study using web-based medical self-management questionnaires.
Methods
Questionnaires consisting of 54 questions in seven domains were filled out by the adolescents before the first shared appointment with both paediatric and adult endocrinologist.
Results
Fifty-seven patients (median age 17 years, 25/57 females) participated and generally scored well on most items. However, one out of seven did not know the name of their disorder, one sixth of the glucocorticoid users did not know that dose should be adapted in case of illness or surgery, over one-fifth had never ordered their repeat prescriptions themselves and two-thirds had never had a conversation alone with their doctor.
Conclusions
Several SMS among patients with rEC are insufficient, with regard to medical knowledge, practical skills and communication. As SMS are only weakly related to non-modifiable factors, such as age and gender, we recommend focussing on other factors to increase transition readiness. The timing, amount and ‘mode’ of medical information should be individualised. Transition checklists should be used to detect shortcomings in practical skills and communication, which can subsequently be trained with the help of parents, caregivers and/or e-technology.
Introduction
Up to 50% of young adults with an endocrine disorder is lost to follow up after transfer to adult health care services (1). There is no consensus about the optimal timing of transition (2). Adolescents and young adults (AYA) have indicated that they are transition-ready between 17 and 40 years old, with the majority preferring the age of 18–24 years for transfer (3, 4). This suggests that some AYA might not be fully prepared to take this step, which can lead to disengagement from healthcare. This disengagement from healthcare, or ‘dropout’, can lead to poor adherence to medication and even hospitalisation (5, 6). Therefore, paediatricians recommend starting the transition process much earlier, beginning at the age of 12 (7).
Apart from age, assessment of self-management skills (SMS) is a good indicator of transition readiness (8). Medical self-management is defined as ‘maintaining satisfaction in living with a chronic disorder through managing the symptoms, medical care, and physical, physiological and social effects associated with the disorder’ (9). Self-management includes know-how of the disease and medication, adherence to medication and a healthy lifestyle, problem solving and symptom management (9, 10). Understanding which factors increase SMS (and thus transition readiness) might help prevent part of dropout.
Up to now, SMS and transition readiness have been mainly studied among AYA with non-endocrine diseases such as inflammatory bowel disease, sickle cell disease, kidney diseases or common endocrine disorders such as diabetes mellitus (5, 11, 12, 13, 14, 15, 16, 17, 18, 19, 20, 21). For example, Monaghan et al. (22) provide an overview of best practices for health care professionals working with adolescents with diabetes type I who are ready for transition. ‘Stimulating patient’s autonomy’ and ‘schedule joint visits with paediatric and adult endocrinologists’ are two examples of best practices recommended by Monaghan et al.
Information about transition readiness in rare endocrine conditions (rEC) is scarce. To minimise dropout after transfer to adult endocrine healthcare, it is essential to examine this particular group of patients in detail. Thus, we aimed to assess medical SMS among AYA with rEC and relate them to patient characteristics, in order to understand and improve transition readiness. To achieve this, we analysed the answers to a web-based survey that patients filled out before visiting the Rotterdam Young Adults Clinic (YAC), a multidisciplinary transition clinic for (young) adults with rare endocrine disorders.
Methods
This observational study was approved by the local ethical review board of the Erasmus University Medical Centre. Consent was obtained from all participants.
Patient selection
The survey was conducted in the Erasmus University Medical Centre, the Netherlands, prior to the first shared appointment with both paediatric and adult endocrinologist at the YAC. Before the implementation of the YAC, no structured transition program was available for patients with rEC. Since 2017, adolescents and young adults with rEC visit the YAC before their first appointment at the adult outpatient clinic. Filling out the SMS questionnaire before the first visit to the YAC is part of regular patient care. Patients do not receive any other questionnaires or written transition support prior to this first visit. For this study, we retrospectively analysed all questionnaires filled out between May 2017 and October 2019. We included questionnaires of patients who were diagnosed with rEC and were able to fill out the questionnaire online without the help of parents or caregivers. A minimum age of 15 years was required to participate. No maximum age was defined, as only patients of paediatric endocrinologists could participate. Patients diagnosed with diabetes mellitus were not included as the Diabetes Care and Research Centre ‘Diabeter’ takes care of these patients outside the hospital setting.
Questionnaire
The questionnaire used in this study was based on the questionnaires used in the so-called ‘Ready Steady Go’ transition program (see 'Acknowledgements' for details), which was developed for children and adolescents in the transition phase. In our study, the original questionnaire was adapted for this particular patient group. The original version of the questionnaire is provided as supplementary data (Supplementary The Ready Steady Go transition programme – Go, see section on supplementary materials given at the end of this article). The (web-based) questionnaire consisted of 54 questions classified into 7 domains that encompass a broad spectrum of medical and psychosocial components: (1) medical knowledge, (2) medication (other than glucocorticoids), (3) use of glucocorticoids (if applicable), (4) communication with the hospital, (5) practical issues and logistics, (6) health and lifestyle and (7) self-advocacy. The questions were answered on a 3-point Likert scale: 'yes', 'more or less' and 'no', with 'yes' as the highest (positive) score and 'no' as the lowest score for the SMS. For the questions regarding medication use, the option 'not applicable' could also be chosen if a question did not apply to the participant. The first question: 'I visit the physician because of…' was a multiple-choice question with 13 answer options, including the names of various rEC like Turner syndrome, Klinefelter syndrome, growth hormone deficiency and the options 'I do not know' and 'other'. When choosing the option 'other', patients could specify which condition they had. When the patient did not give any answer to this question, we assumed that patient did not know the name of his/her condition. The self-reported diagnosis was compared to the diagnosis retrieved from medical records.
Statistical analyses
Data were analysed using IBM SPSS Statistics version 24. Participant characteristics (retrieved from medical records) and responses to the questionnaires were summarised with percentages. Associations between all questionnaire items and gender (as assigned at birth) and between all questionnaire items and age were explored using a Mann–Whitney U test or a Spearman’s rho, respectively.
Results
Sixty-four patients visited the YAC, of which 58 patients filled out the questionnaire. One participant was excluded from the analysis because a parent had filled out the questionnaire. The 57 (32M/25F) included patients had 25 different rEC of which pituitary hormone deficiencies (n = 7), Klinefelter syndrome (n = 6), 46, XY DSD (n = 6) and congenital adrenal hyperplasia (n = 5) were the most common (see Table 1 for details). Median age of participants was 17 years (IQR 17.0–18.0, range 15–26 years), 29 patients were younger than 18 years (51%). Age did not differ significantly between males and females: median age of males was 17.0 years (IQR 17.0–18.0, range 15–26 years) and of females 18.0 years (IQR 17.0–18.5, range 17–20 years). Answers to the questionnaires are shown in Tables 2, 3, 4, 5, 6, 7, and 8.
Diagnosis of the participants according to endocrine gland involvement.
| Diagnosis | Prevalence/incidence | nb |
|---|---|---|
| Adrenal | ||
| Classic congenital adrenal hyperplasia | 1:15,000 (incidence) (37) | 5 |
| Addison’s disease | 1:160,000–230,000 (incidence) (38) | 3 |
| Isolated central adrenal insufficiency | 1:200,000a | 1 |
| Cushing’s syndrome | 1:400,000–1,500,000 (incidence) (39) | 1 |
| Thyroid | ||
| Congenital central hypothyroidism | 1:20,000 (incidence) (40) | 1 |
| Hashimoto’s encephalopathy | 1:48,000 (prevalence) (41) | 1 |
| Multinodular goiter | 1:67–1000 (incidence in non-endemic regions) (34) | 1 |
| Gonads | ||
| Klinefelter syndrome | 1:670 (incidence males) (42) | 6 |
| 46 XY DSD | 1:16,000 (incidence females) (43) | 6 |
| Primary male hypogonadism (not Klinefelter syndrome) | Prevalence depends on type (acquired vs non-acquired) and underlying aetiology (37) | 2 |
| Sex chromosome mosaicism (45X/46XY) | 1:1300 (prevalence) (44) | 2 |
| Premature Ovarian Failure | 1:10,000 of women < 30 years old (45) | 2 |
| Hypogonadotropic hypogonadism (including Kallmann syndrome) | 1:10,000 (incidence) (46) | 3 |
| Anterior pituitary | ||
| Combined pituitary hormone deficiency | 1:8000 (prevalence) (47) | 7 |
| Isolated growth hormone deficiency | 1:4000–10,000 (incidence) (48) | 1 |
| Prolactinoma | 1:10,000 (prevalence) (49) | 1 |
| Posterior pituitary | ||
| Diabetes insipidus | 1:25,000 (prevalence) (50) | 1 |
| Other | ||
| CHARGE syndrome | 1:8300–100,000 (incidence) (51) | 1 |
| MEN1 syndrome | 1:40,000 (incidence) (52) | 2 |
| Temple Syndrome | <1:1,000,000a | 2 |
| Familial pseudohypo-parathyroidism | 1:90,000–300,000 (prevalence) (53) | 1 |
| Miller syndrome | <1:1,000,000a | 1 |
| Osteogenesis imperfecta | 1:10,000 (incidence) (54) | 1 |
| Pontine glioma | 1:400,000 (incidence in 0–20 year olds) (55) | 1 |
| Silver–Russell syndrome | 1:30,000–100,000 (prevalence) (56) | 1 |
aRetrieved from www.orpha.net (portal for rare diseases and orphan drugs); bretrieved from medical records.
Medical self-management questionnaire domain: ‘Medical knowledge’.
| Yes, n | More or less, n | No, n | Missing, n | Gender significance | Age significance | |||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Female, 25 | Male, 32 | <18, 29 | ≥18, 28 | Total, 57 | Female, 25 | Male, 32 | <18, 29 | ≥18, 28 | Total, 57 | Female, 25 | Male, 32 | <18, 29 | ≥18, 28 | Total, 57 | ||||
| Gives right name or description of conditiona | 15 | 19 | 23 | 11 | 34 | 1 | 6 | 2 | 5 | 7 | 9 | 7 | 4 | 12 | 16 | 0 | P=0.70 | P≤0.001 |
| I know the name of my condition | 16 | 23 | 21 | 18 | 39 | 4 | 5 | 3 | 6 | 9 | 4 | 4 | 5 | 3 | 8 | 1 | P=0.65 | P=0.79 |
| I can describe my condition | 18 | 16 | 16 | 18 | 35 | 5 | 15 | 11 | 9 | 20 | 1 | 0 | 1 | 0 | 1 | 1 | P=0.11 | P=0.69 |
| I (usually) understand what doctor is saying | 18 | 22 | 20 | 20 | 40 | 6 | 9 | 8 | 7 | 15 | 1 | 1 | 1 | 1 | 2 | 0 | P=0.82 | P=0.92 |
| I know which treatments/operations I have had in the past and why | 16 | 24 | 20 | 20 | 40 | 7 | 5 | 6 | 6 | 12 | 2 | 2 | 2 | 2 | 4 | 1 | P=0.30 | P=0.98 |
| I understand what is likely to happen with my condition when I am an adult | 9 | 15 | 9 | 15 | 24 | 11 | 11 | 14 | 8 | 22 | 5 | 6 | 6 | 5 | 11 | 0 | P=0.51 | P=0.04 |
| I understand the risks for my health if I do not adhere to the agreements I made with my doctor or nurse | 19 | 19 | 17 | 21 | 38 | 3 | 7 | 6 | 4 | 10 | 2 | 5 | 5 | 2 | 7 | 2 | P=0.16 | P=0.18 |
n, number of participants; acompared to actual disorder as displayed in Table 1. Bold indicates statistical significance.
Medical self-management questionnaire domain: ‘Medication’.
| Yes, n | More or less, n |
No, n | Missing, n | Gender significance | Age significance | |||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Female, 21 | Male, 22 | <18, 19 | >18, 24 | Total, 43 | Female, 21 | Male, 22 | <18, 19 | >18, 24 | Total, 43 | Female, 21 | Male, 22 | <18, 19 | >18, 24 | Total, 43 | ||||
| I know what medication I take | 20 | 22 | 19 | 23 | 42 | 1 | 0 | 0 | 1 | 1 | 0 | 0 | 0 | 0 | 0 | 0 | P=0.31 | P=0.71 |
| I understand what my medication is for | 17 | 17 | 12 | 22 | 34 | 3 | 5 | 6 | 2 | 8 | 1 | 0 | 1 | 0 | 1 | 0 | P=0.84 | P=0.005 |
| I know what will happen if I don’t take my medication | 17 | 13 | 12 | 18 | 30 | 2 | 7 | 4 | 5 | 9 | 2 | 2 | 3 | 1 | 4 | 0 | P=0.17 | P=0.09 |
| I know what to do with my medication in case of vomiting or diarrhoea | 8 | 7 | 5 | 10 | 15 | 5 | 2 | 3 | 4 | 7 | 4 | 7 | 7 | 4 | 11 | 0 | P=0.49 | P=0.16 |
| I know the side effects of my medication | 6 | 5 | 3 | 8 | 11 | 7 | 8 | 8 | 7 | 15 | 7 | 6 | 7 | 6 | 13 | 0 | P=1 | P=0.06 |
| I know what to do if I (think I) experience side effects of my medication | 9 | 3 | 3 | 9 | 12 | 2 | 4 | 3 | 3 | 6 | 8 | 9 | 9 | 8 | 17 | 0 | P=0.19 | P=0.06 |
| I know what to do in case I have forgotten my medication | 18 | 11 | 13 | 16 | 29 | 1 | 5 | 5 | 1 | 6 | 1 | 5 | 0 | 6 | 6 | 2 | P=0.01 | P=0.92 |
| I know how I can recognise that my medication is not well regulated | 9 | 10 | 8 | 11 | 19 | 4 | 4 | 5 | 3 | 8 | 6 | 8 | 6 | 8 | 14 | 1 | P=0.82 | P=0.57 |
| I order my repeat prescriptions myself when my medication has almost run out | 16 | 11 | 10 | 17 | 27 | 2 | 1 | 2 | 1 | 3 | 3 | 8 | 6 | 5 | 11 | 0 | P=0.11 | P=0.14 |
| I know I have to order new repeat prescriptions and collect these in time at the pharmacy | 19 | 16 | 12 | 23 | 35 | 1 | 3 | 3 | 1 | 4 | 0 | 2 | 2 | 0 | 2 | 0 | P=0.09 | P=0.02 |
| I collect my medication at the pharmacy by myself | 16 | 13 | 13 | 16 | 29 | 2 | 6 | 2 | 6 | 8 | 1 | 3 | 3 | 1 | 4 | 0 | P=0.09 | P=0.30 |
| I remind myself to take my medication | 17 | 17 | 13 | 21 | 34 | 4 | 5 | 6 | 3 | 9 | 0 | 0 | 0 | 0 | 0 | 0 | P=0.77 | P=0.10 |
| I know how to store my medication (e.g. In fridge) | 19 | 20 | 17 | 22 | 39 | 1 | 1 | 2 | 0 | 2 | 1 | 1 | 0 | 2 | 2 | 0 | P=0.96 | P=0.62 |
| I know how to use my medication (time, simultaneously with food or not, washing hands after use or not) | 19 | 21 | 17 | 23 | 40 | 1 | 0 | 1 | 0 | 1 | 1 | 0 | 0 | 1 | 1 | 0 | P=0.15 | P=0.69 |
n, number of participants. Bold indicates statistical significance.
Medical self-management questionnaire domain: ‘Glucocorticoid use’.
| Yes, n | More or less, n | No , n | Totala, n | Gender significance | Age significance | |||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Female, 6 | Male, 11 | <18, 8 | ≥18, 9 | Total, 17 | Female, 6 | Male, 11 | <18, 8 | ≥18, 9 | Total, 17 | Female, 6 | Male, 11 | <18, 8 | ≥18, 9 | Total, 17 | ||||
| If I am ill or I have to undergo surgery, I know how to change my hydrocortisone dosage | 2 | 8 | 4 | 6 | 10 | 3 | 1 | 1 | 3 | 4 | 1 | 2 | 3 | 0 | 3 | 17 | P=0.23 | P=0.29 |
| I know what can happen if I do not change the hydrocortisone dosage in these situations | 4 | 5 | 4 | 5 | 9 | 1 | 3 | 1 | 3 | 4 | 1 | 2 | 2 | 1 | 3 | 16 | P=0.59 | P=0.47 |
| People in my direct surroundings know: | ||||||||||||||||||
| – that I have a hydrocortisone injection in case of emergencies when I am not able to take my medication by myself or keep in (e.g. when vomiting or unconscious) | 4 | 7 | 4 | 7 | 11 | 1 | 2 | 1 | 2 | 3 | 1 | 1 | 2 | 0 | 2 | 16 | P=0.84 | P=0.32 |
| – what to do in such a situation | 3 | 7 | 2 | 8 | 10 | 2 | 1 | 2 | 1 | 3 | 1 | 2 | 3 | 0 | 3 | 16 | P=0.57 | P=0.03 |
| I always carry an emergency vial of hydrocortisone and an injection needle with me | 3 | 3 | 1 | 5 | 6 | 1 | 3 | 1 | 3 | 4 | 2 | 4 | 5 | 1 | 6 | 16 | P=0.56 | P=0.051 |
| I wear an S.O.S. bracelet, medallion or tattoo which says that I use hydrocortisone | 3 | 4 | 2 | 5 | 7 | 0 | 1 | 0 | 1 | 1 | 3 | 5 | 5 | 3 | 8 | 16 | P=0.86 | P=0.41 |
n, number of participants; amissing data are not included, since the questions did not apply to participants who did not use hydrocortisone. Bold indicates statistical significance.
Medical self-management questionnaire domain: ‘Communication with the hospital’.
| Yes, n (%) | More or less, n (%) | No, n (%) | Missing, n (%) | Gender significance | Age significance | |||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Female, 21 | Male, 22 | <18, 19 | >18, 24 | Total, 43 | Female, 21 | Male, 22 | <18, 19 | >18, 24 | Total, 43 | Female, 21 | Male, 22 | <18, 19 | >18, 24 | Total, 43 | ||||
| I know why I have regular blood checks | 20 | 28 | 24 | 24 | 48 | 4 | 0 | 2 | 2 | 4 | 1 | 3 | 2 | 2 | 4 | 1 | P=0.36 | P=0.98 |
| I know why I have regular appointments with the doctor | 22 | 30 | 26 | 26 | 52 | 2 | 1 | 1 | 2 | 3 | 1 | 0 | 1 | 0 | 1 | 1 | P=0.20 | P=0.74 |
| I know what will change after my transfer to adult endocrinology | 5 | 7 | 5 | 7 | 12 | 10 | 9 | 10 | 9 | 19 | 10 | 15 | 13 | 12 | 25 | 1 | P=0.72 | P=0.26 |
| I arrange and keep track of doctor’s appointments | 21 | 22 | 19 | 24 | 43 | 1 | 6 | 5 | 2 | 7 | 3 | 3 | 4 | 2 | 6 | 1 | P=0.34 | P=0.03 |
| I arrange my own transport to doctor’s appointments | 15 | 19 | 15 | 19 | 34 | 3 | 6 | 5 | 4 | 9 | 7 | 6 | 8 | 5 | 13 | 1 | P=0.75 | P=0.22 |
| I ask the doctor or nurse questions | 15 | 19 | 16 | 18 | 34 | 7 | 8 | 7 | 8 | 15 | 3 | 4 | 5 | 2 | 7 | 1 | P=.96 | P=.33 |
| I tell the doctor or nurse what I feel and think | 18 | 23 | 20 | 21 | 41 | 6 | 6 | 6 | 6 | 12 | 1 | 2 | 2 | 1 | 3 | 1 | P=0.91 | P=0.54 |
| I see my doctor by myself for some time at every appointment | 4 | 5 | 2 | 7 | 9 | 6 | 3 | 3 | 6 | 9 | 15 | 23 | 23 | 15 | 38 | 1 | P=0.36 | P=0.002 |
| I adhere to the agreements I make with my doctor or nurse (medication, blood checks, lifestyle, etc.) | 20 | 26 | 21 | 25 | 46 | 5 | 5 | 7 | 3 | 10 | 0 | 0 | 0 | 0 | 0 | 1 | P=0.71 | P=0.26 |
| If my condition worsens, I know how to get help | 16 | 20 | 16 | 20 | 36 | 6 | 4 | 6 | 4 | 10 | 3 | 7 | 6 | 4 | 10 | 1 | P=0.80 | P=0.04 |
| I know how to contact (telephone or e-mail) my endocrinologist/internist and/or the outpatient clinic if that is necessary | 14 | 17 | 16 | 15 | 31 | 6 | 2 | 5 | 3 | 8 | 5 | 12 | 7 | 10 | 17 | 1 | P=0.51 | P=0.66 |
| I know how to reach the doctor or nurse outside office hours if necessary | 4 | 4 | 4 | 4 | 8 | 4 | 4 | 1 | 7 | 8 | 17 | 23 | 23 | 17 | 40 | 1 | P=0.61 | P=0.04 |
n, number of participants. Bold indicates statistical significance.
Medical self-management questionnaire domain: ‘Practical issues and logistics’.
| Yes, n | More or less, n | No, n | Missing, n | Gender significance | Age significance | |||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Female, 21 | Male, 22 | <18, 19 | >18, 24 | Total, 43 | Female, 21 | Male, 22 | <18, 19 | >18, 24 | Total, 43 | Female, 21 | Male, 22 | <18, 19 | >18, 24 | Total, 43 | ||||
| I know my health insurance details (policy number, contact/information) | 12 | 19 | 13 | 18 | 31 | 8 | 6 | 7 | 7 | 14 | 5 | 7 | 9 | 3 | 12 | 0 | P=0.56 | P=0.07 |
| I am aware of the consequences of my condition for my insurance | 1 | 7 | 2 | 6 | 8 | 4 | 4 | 3 | 5 | 8 | 20 | 21 | 24 | 17 | 41 | 0 | P=0.16 | P=0.02 |
| I know how to plan ahead for being away from home or having a sleepover (medication, prescriptions and materials) | 24 | 24 | 23 | 25 | 48 | 1 | 3 | 3 | 1 | 4 | 0 | 4 | 2 | 2 | 4 | 1 | P=0.045 | P=0.22 |
| I know how to plan ahead for going abroad (medication, vaccinations, insurance, letter of medical necessity, prescriptions and materials) | 19 | 17 | 17 | 19 | 36 | 5 | 5 | 5 | 5 | 10 | 1 | 9 | 6 | 4 | 10 | 1 | P=0.045 | P=0.08 |
n, number of participants. Bold indicates statistical significance.
Medical self-management questionnaire domain: ‘Health and lifestyle’.
| Yes, n | More or less, n | No, n | Missing, n | Gender significance | Age significance | |||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Female, 21 | Male, 22 | <18, 19 | >18, 24 | Total, 43 | Female, 21 | Male, 22 | <18, 19 | >18, 24 | Total, 43 | Female, 21 | Male, 22 | <18, 19 | >18, 24 | Total, 43 | ||||
| I understand the importance of a healthy lifestyle (exercising, food, etc.) for my condition and overall wellbeing | 20 | 26 | 21 | 25 | 46 | 5 | 4 | 7 | 2 | 9 | 0 | 2 | 1 | 1 | 2 | 0 | P=1 | P=0.03 |
| I understand the effect of smoking, drugs or alcohol on my condition and general health | 22 | 21 | 19 | 24 | 43 | 2 | 8 | 7 | 3 | 10 | 1 | 3 | 3 | 1 | 4 | 0 | P=0.06 | P=0.08 |
| My friends know what condition I have | 13 | 9 | 14 | 8 | 22 | 7 | 12 | 8 | 11 | 19 | 5 | 11 | 7 | 9 | 16 | 0 | P=0.08 | P=0.38 |
| I am in a relationship (or have been) | 12 | 18 | 15 | 15 | 30 | 1 | 2 | 2 | 1 | 3 | 12 | 12 | 12 | 12 | 24 | 0 | P=0.48 | P=.59 |
| I understand the implications of my condition and medication on sexuality and possible pregnancy | 11 | 14 | 12 | 13 | 25 | 5 | 8 | 3 | 10 | 13 | 9 | 10 | 14 | 5 | 19 | 0 | P=0.86 | P=0.03 |
| If I have questions about sexuality, I have someone I can talk to If I would like (e.g. my parents, doctor or nurse) | 20 | 24 | 23 | 21 | 44 | 4 | 6 | 4 | 6 | 10 | 1 | 1 | 1 | 1 | 2 | 1 | P=0.84 | P=0.55 |
| I know how to avoid situations like unwanted pregnancies or sexually transmitted diseases (STDs) | 24 | 27 | 26 | 25 | 51 | 1 | 3 | 2 | 2 | 4 | 0 | 2 | 1 | 1 | 2 | 0 | P=0.15 | P=0.49 |
n, number of participants. Bold indicates statistical significance.
Medical self-management questionnaire domain: ‘Self advocacy’.
| Yes, n | More or less, n | No, n | Missing, n | Gender significance | Age significance | |||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Female, 21 | Male, 22 | <18, 19 | >18, 24 | Total, 43 | Female, 21 | Male, 22 | <18, 19 | >18, 24 | Total, 43 | Female, 21 | Male, 22 | <18, 19 | >18, 24 | Total, 43 | ||||
| I know my rights and duties concerning my medical treatment (supply of information, privacy, decision-making) | 11 | 12 | 9 | 14 | 23 | 8 | 8 | 10 | 6 | 16 | 6 | 11 | 10 | 7 | 17 | 1 | P=0.48 | P=0.02 |
| I know which organisations support adolescents with my condition | 5 | 3 | 4 | 4 | 8 | 3 | 5 | 2 | 6 | 8 | 17 | 23 | 23 | 17 | 40 | 1 | P=0.51 | P=0.22 |
| I know the possible consequences of my condition for my education and future job | 10 | 16 | 12 | 14 | 26 | 1 | 5 | 5 | 1 | 6 | 14 | 10 | 12 | 12 | 24 | 1 | P=0.17 | P=0.27 |
| I know how and what to tell a (potential) employer or mentor about my condition | 15 | 17 | 16 | 16 | 32 | 5 | 10 | 10 | 5 | 15 | 5 | 4 | 3 | 6 | 9 | 1 | P=0.93 | P=0.62 |
n, number of participants. Bold indicates statistical significance.
Results per domain
Medical knowledge
One out of seven patients did not know the name of their disorder. One-sixth stated they knew the name ‘more or less’. Two-thirds of the participants were confident that they knew exactly what diagnosis they had. Three out of five patients indeed gave the correct name or description (Table 2).
Medication
All AYA knew what kind of medication they were using and 34/57 knew what they were using it for. Almost half of the patients ordered their own repeat prescriptions, but one-fifth fully relied on their parents or caregivers for this. About one-fifth of the patients were aware of the potential side effects of the medication they were taking (Table 3).
Glucocorticoid use
Of the glucocorticoid (GC) users (n =17), one fifth did not know that GC dose should be adjusted in case of stress, illness or surgery. They were unaware of what could happen if the GC dosage was not increased in this situation. Half of the patients did not wear an SOS item like bracelet or medallion. One-third did not always carry an emergency vial of GC with them (Table 4).
Communication with the hospital
Two-thirds of patients never talked to the doctor without the presence of parents or caregivers. 25/56 of the patients did not know what changes they could expect after transfer to adult healthcare (Table 5).
Practical issues
41/57 patients did not know the consequences of their condition for their health insurance (Table 6).
Health and lifestyle
Nine out of ten patients knew the effects of smoking, drugs and alcohol on their condition. Two-thirds understood the implications of their condition and medication on sexuality and possible pregnancy. Two-thirds had informed friends about their disorder (Table 7).
Self-advocacy
Two-thirds of the AYA knew their rights and duties concerning medical treatment. However, almost three-quarters of the patients did not know whether patient support organisations existed for patients with their condition (Table 8).
Relation with gender
Females were better informed about certain items regarding medication: what to do when having forgotten medication (P = 0.01) (Table 3) and what to do with medication when travelling (P = 0.045) (Table 6).
Relation with age
Younger patients scored significantly higher on giving the right name or description of their condition (P < 0.001; Table 2). Older patients more often knew what their medication was for (P < 0.005; Table 3). Older patients more often talked to the doctor without their parents present (P < 0.002; Table 5). Compared with younger patients, older patients did not score higher on knowing how to change GC dosages in case of emergency (P = 0.29; Table 4) or how to contact their endocrinologist if necessary (P = 0.66; Table 5). There were some borderline significant age differences (0.01 < P < 0.05) for other items (Tables 2, 3, 4, 5, 6, 7 and 8).
Discussion
We studied transition readiness among 57 adults and young adults with rare endocrine disorders, using a web-based self-management questionnaire. In general, patients scored well on all self-management items which means that, for the majority of the patients, on the majority of the self-management items, answers were positive. SMS were only weakly related to non-modifiable factors, such as age and gender, which is in accordance with previous research (21, 23). As SMS correlate poorly with these non-modifiable factors, we looked at possible ways to improve modifiable factors, like filling gaps in medical information or providing instructions about practical issues (e.g. changing GC dosages in case of emergency or contacting the endocrinologist if necessary).
Medical information
One out of seven patients did not know the name of their disorder. Likewise, one out of seven patients only knew the name of their disorder ‘more or less’. Although some disorders with difficult names like 5-alpha reductase deficiency could be harder to remember than disorders like Klinefelter syndrome, this might suggest that almost one-third of the patients are not sufficiently informed about their condition at the moment of transfer to adult healthcare. This is confirmed by the answers to the question 'I can describe my condition' which was answered affirmatively by only 61%. This suboptimal knowledge of the medical condition contrasts with what was found in patients with thalassemia and diabetes mellitus, where the majority could name their condition and medication (3). According to patients, medical knowledge is an important factor in transition readiness (11). This was confirmed in a systematic review by Stinson et al. (21), which showed that medical knowledge is positively related to transition readiness. Medical knowledge is therefore an important target for improving transition readiness and should also be optimised in patients with rEC. Apart from improving medical knowledge by optimising the timing, amount and ‘modality’ (spoken, written or video) of medical information, an informative website or smartphone app might also be useful.
Practical issues
Self-management regarding the use of medication in day-to-day life was generally well developed among the adolescents. Yet, situations that deviate from a regular day (e.g. changing GC dosages in case of emergency or contacting their endocrinologist if necessary) were generally considered challenging and this was independent of age. This is in line with a recent study by Lau et al. (3) and suggests that AYA might find it difficult to adapt to non-standard medical circumstances. In our hospital, all patients who use GC receive a hydrocortisone stress scheme along with the contact details of their doctor. Moreover, important information regarding GC use is often repeated to increase adherence. Surprisingly, we found that many patients still do not know when to adjust their dosage (and why) and that almost two-fifths do not carry an emergency vial of GC. Half of the patients do not wear an S.O.S bracelet, medallion or tattoo, even though this is strongly recommended (8, 24). Not all information given to patients, provided by their physician, is remembered (25). Even patients that suffer from a chronic disease, can only partly recall information (26). Apart from giving oral instructions and written information, other approaches could help to remember information. Examples include visual aids and playful learning tools (gamification) which could help to optimise medical knowledge and adherence to medical advice (25, 27). Furthermore, involving parents could also be beneficial to patients undergoing transition. In a review conducted by Tully et al. (28, 29), coaching parents from children with type I diabetes seemed to be an easy intervention in providing psychosocial support. Although a younger age group was studied, coaching parents of (young) adults with rEC might also be helpful.
Patients were generally unaware of the possible implications of their disorder for health insurances, job searching, sexuality and possible pregnancies. In addition, our data show that the minority knew what changes they could expect after transfer to adult healthcare, which was in line with previous findings in a tertiary paediatric department (3). However, this is not a major concern as this subject is thoroughly discussed at a later stage during the visits at the YAC. Once the patient visits the YAC, the treating physician explains the differences between the paediatric and adult healthcare, along with what the patient can expect from the new adult endocrinologist and what is expected from the patient (e.g. in terms of taking responsibility and making their own decisions). Furthermore, our data demonstrate that knowledge about sexuality, pregnancy and healthcare insurances is better in older subjects, which is reassuring as it suggests that these topics will be learnt over time.
Communication and other
SMS AYA were reasonably independent in terms of planning appointments and visiting the doctor. However, they could be more independent regarding communication with the doctor. Two-thirds of the patients had never had a conversation with the doctor by themselves. This should be further improved during the transition period. If the training of communication with the doctor is not encouraged, patients might lag behind in terms of assertiveness and taking responsibility in managing their own health issues. Therefore, we recommend the use of transition checklists to detect these ‘shortcomings’. Examples of well-validated checklists are TRAQ (Transition Readiness Assessment Questionnaire) (30) and TRAM (Transition Readiness and Appropriateness Measure) (31). It is important to detect these ‘shortcomings’ in time, so that patients have enough time to train all skills prior to transfer.
The gaps in SMS detected by transition checklists can subsequently be trained with the help of parents and caregivers, provided that both patient and parents are motivated to do so. Suris et al. (32) and Roth et al. (23) found that patients are not always motivated to learn how to be more independent. If patients are less motivated, a transition app can help to make the transition easier, and serious gaming (33, 34, 35) can be a way to make self-management fun. This way, e-technology can help the patient to become more independent.
Like every study, our study has strengths and limitations. The major strength is the unique population of patients with rare disorders. Limitations of our study include confined generalisability and small sample size. Our participants may not be representative for all AYA with rEC, as enrolment took place in one medical centre. Furthermore, social desirability could have influenced the answers, leading to an overestimation of SMS (36). Moreover, as the patients filled out the questionnaires at home, we cannot be certain that they did not receive help from parents or caregivers. Nonetheless, this is the first study, to our knowledge, that has assessed an extensive variety of self-management items among AYA with rEC.
In conclusion, we assessed transition readiness among 57 adolescents and young adults with rare endocrine disorders. In general, patients scored well on all self-management items. However, there is room for improvement of SMS, with regard to knowledge of the disorder, practical issues (like dealing with unexpected situations, collecting repeat medication, healthcare insurance) and communication with the hospital. SMS were only weakly related to non-modifiable factors like age or gender. Therefore we recommend focusing on other factors to improve transition readiness. First, the timing, amount and ‘mode of administration’ (i.e. spoken, written, e-technology) of medical information should be individualised and repeated. Secondly, transition checklist should be used to actively search for gaps in SMS. Thirdly, detected ‘shortcomings’ in communication and other SMS can be trained with the help of parents, caregivers or e-technology (apps and/or serious games). These measures require relatively little effort and can help AYA bridge the gap between paediatric and adult endocrinology.
Supplementary materials
This is linked to the online version of the paper at https://doi.org/10.1530/EC-20-0304.
Declaration of interest
The authors declare that there is no conflict of interest that could be perceived as prejudicing the impartiality of the research reported.
Funding
This research did not receive any specific grant from any funding agency in the public, commercial or not-for-profit sector.
Acknowledgements
The authors made use of '"Ready Steady Go" and "Hello to adult services" developed by the Transition Steering Group led by Dr Arvind Nagra, paediatric nephrologist and clinical lead for transitional care at Southampton Children’s Hospital, University Hospital Southampton NHS Foundation Trust. This was based on the work of (1) S Whitehouse and MC Paone. Bridging the gap from youth to adulthood. Contemporary Pediatrics; 1998, December 13–16. (2) Paone MC, Wigle M, Saewyc E. The ON TRAC model for transitional care of adolescents. Prog Transplant 2006;16:291–302. (3) Janet E McDonagh et al., J Child Health Care 2006;10(1):22–42'. Further information can be found at www.uhs.nhs.uk/readysteadygo.
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