Despite improved health care standards and better understanding of pathophysiology, systemic sclerosis (SSc) has an increased mortality and high morbidity. Management of SSc can be challenging due to its heterogeneous nature, multi-organ involvement and limited therapeutic options. This thesis zooms in on organisation of health care and autologous stem cell transplantation (SCT) as potential targets to improve outcomes.
The organization of Dutch SSc care was investigated and evaluated from different perspectives. Ways to improve quality of care were explored.
Our large cross-sectional study including 650 Dutch SSc patients from thirteen hospitals, showed that patients are generally satisfied with their care. Most important points for improvement from the patient' perspective were education, clear and transparant definitions of SSc expert centers accesible for patients, and collaboration between doctors. Also, patients prioritized good patient-doctor interaction and structural multidisciplinary collaboration as the most relevant quality indicators for SSc care.
In addition to the perspective of patients on health care organization, recommendations were formulated in a multidisciplinary study among doctors, nurses and paramedics. Five domains in need for improvement (shared care and multidisciplinary collaboration, exchange of medical data, education, patient empowerment and non-pharmacological care) were identified and discussed in this meeting.
Furthermore, in order to support collaboration and referral structures on a regional and national level, a Delphi consensus study was done, resulting in requirements for two types of SSc centres with different levels of expertise.
Another neglected area in SSc care is the perspective of caregivers. In an exploratory study the impact of SSc on caregivers and their unmet needs are described. Caregivers reported that their relationship, social lives and emotional wellbeing were affected by the disease of their loved-one. Moreover, supporting their partner or friend was sometimes hindered by a lack of knowledge about the disease, insight in the overall treatment strategy and skills to optimise communication.
The importance of support and information for patients, also provided by health care professionals and peers was emphasized in this thesis. This is particularly true in the context of a complex treatment modality such as SCT. SCT is increasingly used in severe form of SSc. This treatment has demonstrated to greatly improve long-term outcomes in the diffuse subset of SSc. However, treatment-related mortality is relatively high. In the absence of strong evidence on the exact place of SCT in the course of the disease, treatment decision-making rests on the shoulders of patients and their doctors. Patient reported that this process can be very difficult and strongly guided by the physician.
Also, a study on the experiences of patients during and after SCT showed that this therapy has a major physical and psychosocial impact, and that post-treatment counseling should be offered pro-actively. Lastly, in order to optimise outcomes, more research is needed to determine the optimal treatment strategy. The international UPSIDE study aims to provide more insight in optimal timing and patient selection with regard to SCT and immunosuppressive therapy. The study design is described in this thesis.