Mapping of the current transition of care practice for patients with pituitary disease at Endo-ERN reference centers

Background

Disorders of the pituitary gland can affect individuals of any age or sex, depending on the type of the disease (1, 2). Diagnoses include neoplasms of the pituitary fossa, for example, craniopharyngiomas, Rathke’s cleft cysts, or functioning and non-functioning pituitary adenomas (1, 2, 3), and congenital or acquired hypopituitarism (defined by either a partial or a complete deficiency of pituitary hormone secretion) (4). Other ultra-rare manifestations of pituitary diseases include pituitary carcinomas, autoimmune disorders, and vascular events (e.g. apoplexy) (3). Due to the differences in age-related prevalence, the adult endocrinologist has generally more expertise in pituitary adenoma and the pediatric endocrinologist in congenital forms and craniopharyngiomas, so shared care is relevant to ensure optimal expertise.

The management of these conditions requires special precautions during the most delicate phases of patients' lives, for example, in the transition from childhood to adulthood. Despite the importance of this period, agreement on how to define (timing of) transition is nonexistent. Previously, physicians have defined transition as the transitional phase between the end of puberty and reaching peak bone mass, involving a wide range of physical, psychological, and sociocultural changes (5). For healthcare systems, however, transition is defined as the planned movement of adolescents and young adults with chronic physical and medical conditions with associated needs from child-centered to adult-oriented assistance (6). Several authors have defined the start of transition by physical development, with Tanner stage 5 being the starting point (5, 7), whereas other authors preferred to consider chronological age as a cut-off value (8). Optimal expertise for the disease may also affect the decision about transition. Nevertheless, a uniform definition, specifically for patients affected by pituitary diseases, is needed to improve uniformity and (quality) standards of care.

Currently, a well-structured and evidence-based transition of care protocol for patients with pituitary diseases is lacking worldwide. The commitment of the European Reference Network on Rare Endocrine Conditions (Endo-ERN) (9) is to lay the foundations for standardization of care through guideline conformity. Therefore, the aim of this study was to map the current clinical practice regarding transition of care across reference centers (RCs) of Endo-ERN, which have specifically been endorsed for their expertise on hypothalamic and pituitary conditions, and to identify the needs regarding transition of care for these patients. The need to consider the general transition aspects alongside the pituitary-specific ones is highlighted throughout the current work. However, general transition aspects will not be discussed at length, as respondents are primarily pituitary experts.

Methods

Results

Transition of care practices

Determinants for the start of transition of care

The most influential factors for the start of transition were age (n  = 42/46, 91.3%), personal judgement of the clinician (n  = 25/46, 54.3%), skills of the patient (n  = 22/46, 47.8%), wish of the patient (n  = 21/46, 45.7%), and outcomes (n  = 14/46, 30.4%), as shown in Fig. 1. When asked about the chronological age of start of transition, the most common answer was 16–18 years old (n  = 22/40, 55%), and the least common answer was <14 years (n  = 1/40, 2.5%).

View Full Size

Criteria defining borders of transition period. (A) The bar chart demonstrates the number of respondents that consider each of the factors shown as important for the beginning (blue) and the end (orange) of the transition process. (B) Number of physicians considering the age at transition initiation. (C) Number of physicians considering the age at end of transition. (B) and (C) show the average age at which the respondents usually initiate and end the transition process. Forty of the respondents gave information for the age at start and 20, for the age at end.

Citation: Endocrine Connections 12, 2; 10.1530/EC-22-0308

• Download Figure
• Download figure as PowerPoint slide

Unmet needs and future directions

Results from the survey questions regarding the necessary changes in the current transition practices (using a 5-point Likert scale) are represented in Fig. 2. Key priorities reported were interventions that would enhance patients’ self-management and adherence to treatment and improvement of the collaboration between pediatric and adult endocrinologists. Twelve of the participants pointed out the unfulfilled needs in the transition of care, which included development of a guideline (n  = 5), development of an EU-wide approach (n  = 2), further mapping the needs of the practitioners (n  = 1), sharing currently existing protocols (n  = 1), and definition of successful transition (n  = 1).

View Full Size

Likert scale results. Most needed changes in the current transition practices according to the respondents.

Citation: Endocrine Connections 12, 2; 10.1530/EC-22-0308

• Download Figure
• Download figure as PowerPoint slide

Discussion

The present survey-based study investigated the current practices of transition of care for adolescents with pituitary diseases across Endo-ERN RCs, with the aim to assess the differences in transition management and to identify gaps and unmet needs in the current practices. Major findings were the widespread lack of pituitary-specific transition protocols, heterogeneity of practices, and lack of affirmed measurable outcomes. These issues are acknowledged by clinicians and are perceived as major unmet needs for optimal quality of care for patients with pituitary diseases.

The life phase of transition is difficult to manage because of the many challenges that occur in that period of time, especially when having a chronic endocrine disease (1). Many additional aspects of a patient’s personal life are complicated by the preexisting diseases or its sequels and comorbidities and need special attention to prevent detrimental disruption (15). Therefore, there is a high risk in these patients’ special needs being unmet.

Clearly, the first and most difficult challenge to overcome is the lack of a uniform definition for transition of care. In the present literature, differing definitions reflect many perspectives (vide supra). Based on the survey results, definitions most commonly emphasized the process of transferring care to adult services, the need for planning, and the interdisciplinary approach required to perform the transferring. Interestingly, two of the responders view transition of care as either an administrative burden or as a possibility to improve physicians’ expertise, responses crucial to identifying facilitators and barriers toward a successful transition process (16). Transition of care is certainly complicated by the administrative tasks required for the referral of a patient. Simultaneously, expertise building is an issue for adult endocrinologists, who had to manage pediatric conditions without being accustomed to these age-related conditions and challenges.

Only a few RCs reported that a written transition protocol or ‘guideline’ was currently available, which might be explained by the lack of literature on this topic or lack of uniformity in clinical practice, highlighting the need for transition of care guidelines. In the absence of protocols, responding RCs and healthcare providers have managed transition of care differently. Based on the survey results, transition was most commonly managed by pediatric-adult combined meetings, although these meetings varied in number and frequency. Moreover, the course and timing of transition are mostly based on expert opinion rather than an algorithm. This flexible, personalized approach demonstrated the lack of coherent guidelines for transition of care for pituitary patients, with a clinical practice guideline being a current unmet need. The fact that protocols are under development at a number of RCs indicates that the problem is acknowledged. Ideally, aligned multicenter efforts on a European level would benefit patient care.

At present, specific tools (e.g. questionnaires) to assess the readiness for transition of care are seldom used in the responding RCs, although there are multiple tools available, albeit mostly untailored to pituitary disease (e.g. Ready Steady Go and TRAQ (13, 14)). The only pituitary-specific tool used was the transition readiness checklists and questionnaires by the Endocrine Society, but this tool is not incorporated in a structured transition program, unfortunately for unknown reasons. The overlooking of developed tools is an important observation, as it may be one of the barriers to implementing a transition protocol. The use of generic tools has the advantage of uniformity and comparability between conditions, as well as many psychosocial aspects are in common for chronic illnesses (17, 18). By contrast, a disease-specific tool would offer the possibility to track disease progression and (more) detailed measures of medical management. In one of the RCs where a transition of care protocol is under development, a complete transition of care strategy is applied, combining TRAQ with multidisciplinary transition clinics, electronic reminders, and assessment of PROMs. This integrated approach has been proven beneficial in a recent systematic review (19). Whether the generic, the disease-specific, or a mixed approach is preferable for patients with pituitary diseases should be addressed in the future. Regardless, transition intervention appeared to be cost-effective (20).

Despite the lack of transition protocols, factors influencing the timing of transition are well-known and appreciated by clinicians. Age was the most dominant factor influencing the start of transition. By contrast, age is considered less important for the end of transition. Moreover, medical outcomes and skills of the patient take on a more important role at the end of transition. The average age of transition onset from our survey is higher than that usually cited in the literature (https://www.gottransition.org) (21, 22).

This discrepancy between the theoretical framework and practical application could be explained by administrative requirements or specificity of pituitary population, or societal changes with time, although these questions cannot be answered using the present survey data. Age alone is not a sufficient criterion for appropriate timing. Therefore, establishing a transition ‘window’ is vital prior to the development of protocols or guidelines.

Another prerequisite for successful management of rare diseases – the multidisciplinary team – is an integral part of the transition process. The present observations confirm that the circumstances required for structured transition program may already be present at most of the RCs. Multidisciplinary team meetings combined with pediatric-adult clinics, transfer documentation, and assessment of readiness (with or without a specific tool) are already applied in large proportion of the centers. Despite the fact that most of the prerequisites for structured transition may already be present, multiple decisions were taken based solely on the specialists’ opinion. Beyond the healthcare providers involved, innovative transition models could include alternative clinical environments and methods that may suit young people more such as phone/video appointments, text message communication, or flexible evening consultations (23).

The success of transition programs and interventions in pituitary disease should be explored further, despite the fact that they are proven valuable in other chronic diseases, based on criteria like loss to follow-up or adherence (24, 25). Taking hypopituitarism as an example, certain biochemical, auxological, and psychosocial parameters are widely accepted goals of treatment. In view of the transition process, few of the respondents view medical outcomes as markers for its success, demonstrating lack of understanding that the transition process directly affects medical outcomes and the need for unified criteria for assessment of pituitary transition interventions.

Another important observation is that large proportions of the clinicians do not routinely measure the effects of transition-related interventions, with PROMs being the least employed method of evaluation. PROMs enable putting the patients experience in a context of value and thereby provide additional information on the results and cost-effectiveness of interventions (26). In addition to the traditional medical outcomes, PROMs – and perhaps even patient-reported experience measures (PREMs) – should be implemented in a tailored pituitary transition protocol (27). As there is no unanimity concerning the set of medical (physician-reported) and PROMs, the success of the transition process is not defined, which requires further research as it is of great practical importance.

An alternative to the transition clinics and combined meetings mentioned by one of the RCs is that endocrinologists with expertise in specific diseases take care of the patients independent of age. This is a model in which certain problems of transition (e.g. the fragmentation of services and transfer to new settings and team) are avoided, whereas other medical and organizational problems (e.g. larger administrative staff required) arise. A similar non-transitional model has been described in diabetes care as well (28). Notably, this model of uninterrupted care has not been compared to transition of care model yet. Moreover, this model of care does not reflect the specific expertise required to manage either pediatric or adult patients. Therefore, the current level of knowledge and medical practice indicates that transition of care model might be preferable to tailor to the patients’ needs with the physicians’ expertise.

Following the present exploration of the practice and organization of transition of care in Endo-ERN RCs, multiple important facets have become evident: (a) transition of care is commonplace, mostly planned, and most often done at combined clinics; (b) generic and pituitary-specific (focused on hypopituitarism) transition readiness assessment tools are available but rarely used; (c) transition of care for pituitary disease guidelines are needed; (d) methods to assess success and outcomes of transition are needed, including patient experiences. Translating these findings and clinical expertise, we propose the following model for transition of care in pituitary disease and highlight the most important unmet needs in Fig. 3. Transition of care can be described in five phases: (a) full pediatric care, (b) identification of eligible patients, (c) initiation of readiness assessments, (d) transfer from pediatric to adult care, and (e) full adult care. For all phases, both known and unknown aspects (i.e. unmet needs) were identified, which require further analysis of the landscape and possibilities. Future research should focus on the identification of barriers to the transition process, critical outcomes (both general and pituitary-specific), and the most appropriate measures to measure these outcomes. These are some of the necessary steps that precede any future work on a guideline in the topic. Ideally, such a guideline should be developed by a multinational board taking in consideration evidence-supported patient and expert opinion. In light of the poor standardization of transition practices, such efforts should be a priority for European pituitary experts.

View Full Size

Transition timeline. An optimal timeline of transition based on the survey results. Five major phases are displayed: pediatric care, identification of eligible patients, readiness assessment, transfer to adult care, and full adult care. For each of these phases, appropriate interventions and unmet needs are also shown in the figure.

Citation: Endocrine Connections 12, 2; 10.1530/EC-22-0308

• Download Figure
• Download figure as PowerPoint slide

Several limitations need to be addressed. Multiple biases possibly influence the results of this survey. Selection bias is likely, as not all European countries are represented, and the geographical location of RCs was not distributed equally among countries. Moreover, all respondents are pituitary experts and might therefore overvalue pituitary-specific tools and questionnaires. Finally, twice as many adult endocrinologists participated in the survey, which may have biased the results.

Conclusion

To date, the clinical practice of transition of care for patients with hypothalamic and pituitary conditions varies greatly, even in expert RCs endorsed by Endo-ERN for the diagnosis, treatment, and management of these specific rare conditions. Definitions of successful transition of care and factors facilitating the successful transition should be identified. The proposed model of transition of care for patients with pituitary disease needs to be developed further and systematically assessed in the future.

Supplementary materials

This is linked to the online version of the paper at https://doi.org/10.1530/EC-22-0308.

Declaration of interest

All authors listed have made substantial, direct, and intellectual contribution to the work and approved it for publication.

Funding

This publication is supported by the European Reference Network on Rare Endocrine Conditions (Endo-ERN). Endo-ERN is funded by the European Union within the framework of the EU4H Programme, grant agreement No. 101084921.

Ethics approval and consent to participate

Since no patient data were collected during this study, approval of the Medical Ethical Committee was not required.

Author contribution statement

All authors listed have made substantial, direct, and intellectual contribution to the work, and approved it for publication. The Endo-ERN respondents of Reference centers for MTG6 which are part of the Endo-ERN pituitary transition of care study group. All authors evaluated the study.