French protocol for the diagnosis and management of hematopoietic stem cell transplantation in autoimmune diseases.

Hematopoietic stem cell transplantation (HSCT) for severe ADs was developed over the past 25years and is now validated by national and international medical societies for severe early systemic sclerosis (SSc) and relapsing-remitting multiple sclerosis (MS) and available as part of routine care in accredited center. HSCT is also recommended, with varying levels of evidence, as an alternative treatment for several ADs, when refractory to conventional therapy, including specific cases of connective tissue diseases or vasculitis, inflammatory neurological diseases, and more rarely severe refractory Crohn's disease. The aim of this document was to provide guidelines for the current indications, procedures and follow-up of HSCT in ADs. Patient safety considerations are central to guidance on patient selection and conditioning, always validated at the national MATHEC multidisciplinary team meeting (MDTM) based on recent (less than 3months) thorough patient evaluation. HSCT procedural aspects and follow-up are then carried out within appropriately experienced and Joint Accreditation Committee of International Society for Cellular Therapy and SFGM-TC accredited centres in close collaboration with the ADs specialist. These French recommendations were performed according to HAS/FAI2R standard operating procedures and coordinated by the Île-de-France MATHEC Reference Centre for Rare Systemic Autoimmune Diseases (CRMR MATHEC) within the Filière FAI2R and in association with the Filière MaRIH. The task force consisted of 3 patients and 64 clinical experts from various specialties and French centres. These data-derived and consensus-derived recommendations will help clinicians to propose HSCT for their severe ADs patients in an evidence-based way. These recommendations also give directions for future clinical research in this area. These recommendations will be updated according to newly emerging data. Of note, other cell therapies that have not yet been approved for clinical practice or are the subject of ongoing clinical research will not be addressed in this document.

[Exploring illness perceptions of lupus patients for a motivational environment in therapeutic education]. / Exploration des dimensions de la personne lupique pour un environnement motivationnel en éducation thérapeutique du patient.

PURPOSE: Therapeutic education (TE) intends to help patients with systemic lupus erythematosus to better understand their disease and to improve their quality of life. The objective of this study was to assess illness perceptions of the person to provide a motivational environment for TE. METHODS: Systemic lupus erythematosus patients followed in the department of internal medicine in Lille university hospital responded to a questionnaire assessing five dimensions of the person as proposed by Giordan: the cognitive (knowledge), perceptual (fatigue and pain), affective (anxious and depressive symptoms), infra-cognitive (intimate reasoning) and metacognitive (worldview) dimensions. The quality of life was also evaluated. RESULTS: One hundred and twenty-four patients (114 women (92%); mean age 44.3±14.3 years) responded to the questionnaire. Regarding the cognitive dimension: quantity of information at the time of diagnosis was considered insufficient for 57 patients (46%). The median adherence evaluated by a scale had a median 97mm [88-100]. Regarding the perceptual dimension: pain was assessed at 59mm [44-78] and fatigue at 66mm [50-79] at visual scales. Regarding the affective dimension: prevalence of anxiety symptoms was 67% (83/124) and 28% (35/124) for depressive symptoms. Regarding the infra-cognitive dimension 78 patients (63%) had an external control place. Concerning the metacognitive dimension, systemic lupus erythematosus had repercussions on professional and family life. The quality of life was impacted. CONCLUSION: Multiple dimensions of systemic lupus erythematosus patient have to be considered for an optimal motivational environment for the practice of TE.

[Complaints, needs of patients with systemic sclerosis: a better understanding for a better care]. / Plaintes et besoins des patients atteints de sclérodermie systémique : une meilleure connaissance afin d'améliorer le suivi.

PURPOSE: Systemic sclerosis (ScS) is very heterogeneous in its clinical presentation and its therapeutic care is not codified. A better knowledge of the patients' needs and complaints could improve the patient educational strategies and their global care. METHODS: A self-administered questionnaire aimed to the ScS patient was developed by subspecialty physicians and nurses involved in patient education. It was a cross-sectional study that also included several validated scales: the health control locus scale, the Mactar, HAD and sHAQ scales. RESULTS: One hundred and eight patients (91 women; 18 limited ScS, 71 limited cutaneous ScS, 19 diffuse ScS) filled in the questionnaires. Fatigue was the main complaint in all types of ScS, independently of the ScS type. The aesthetic discomfort mentioned by the patients suffering from cutaneous sclerosis or from telangectasia was important and reached 52±33mm on a 100-mm visual scale. It was more common in the patients presenting a diffuse form of the illness but the difference did not reach a statistical significance (P=0.06). Twenty-seven percent of the patients said they were very or extremely worried because of the degradation of their physical appearance. The functional discomfort linked to the cutaneous sclerosis was rated 50±32mm on a 100-mm visual scale. The intensity of the pain, the importance of the functional discomfort linked to the sclerosis and the intensity of the dyspnea were correlated to the sHAQ (P<0.001). Patients having more frequent recurrent digital ulcers had higher sHAQ scores (P=0.04). The repercussions on the professional life were linked to fatigue first, to the Raynaud's syndrome and to arthralgia. The repercussions on the personal life were mainly linked to the fatigue, the pain and the dyspnea. The patients' compliance was good. CONCLUSION: Fatigue, pain, dyspnea and discomfort linked to sclerosis are major chronic symptoms of the patients with ScS. Identifying the needs and complaints of the patients with ScS should help to improve their care by implementation of an educational program.

[Economic evaluation of at home subcutaneous and intravenous immunoglobulin substitution]. / Etude comparative du coût du traitement à domicile des immunoglobulines intraveineuses ou sous-cutanées à visée substitutive.

BACKGROUND: Intravenous (IV) and subcutaneous (SC) immunoglobulin (Ig) administration is a safe and an efficacious treatment in patients with IgG deficiency. Home administration of IV Ig and SC Ig has recently been approved in France. Most of the patients treated in hospital ask for home treatment. Some patients prefer monthly IV administration, others weekly SC administration. MATERIAL AND METHODS: We evaluated in details the cost of both of these practices. We currently use electric pumps for both IV and SC administration which are fixed to the patient giving complete freedom and mobility. RESULTS: For 20 g administrated per 4 week, the total cost of home treatment is 1,518 euro with SC Ig and 1,033 euro with IV Ig. For 40 g administrated per 4 week, the total cost of home treatment is 2,507 euro to 2,729 euro with SC Ig and 2,034 euro with IV Ig. The difference is mainly explained by the cost of renting the electric pumps and by the cost of furniture for SC administration. The choice of home Ig substitution must be given to all patient receiving treatment in hospital. CONCLUSIONS: Home IV and home SC perfusions are two possible options each different with there own advantages and disadvantages. The cost of each procedure must be known by the medical staff.