Recessive dystrophic epidermolysis bullosa (RDEB) is an incurable inherited mucocutaneous fragility disorder characterized by recurrent blisters, erosions, and wounds. Continuous blistering triggers overlapping cycles of never-ending healing and scarring commonly evolving to chronic systemic inflammation and fibrosis. The systemic treatment with allogeneic mesenchymal cells (MSC) from bone marrow has previously shown benefits in RDEB. MSC from adipose tissue (ADMSC) are easier to isolate. This is the first report on the use of systemic allogeneic ADMSC, correlating the clinical, inflammatory, and immunologic outcomes in RDEB indicating long-lasting benefits. We present the case of an RDEB patient harboring heterozygous biallelicCOL7A1gene mutations and with a diminished expression of C7. The patient presented with long-lasting refractory and painful oral ulcers distressing her quality of life. Histamine receptor antagonists, opioid analgesics, proton-pump inhibitors, and low-dose tricyclic antidepressants barely improved gastric symptoms, pain, and pruritus. Concomitantly, allogeneic ADMSC were provided as three separate intravenous injections of 10⁶cells/kg every 21 days. ADMSC treatment was well-tolerated. Improvements in wound healing, itch, pain and quality of life were observed, maximally at 6–9 months post-treatment, with the relief of symptoms still noticeable for up to 2 years. Remarkably, significant modifications in PBL participating in both the innate and adaptive responses, alongside regulation of levels of profibrotic factors, MCP-1/CCL2 and TGF-β, correlated with the health improvement. This treatment might represent an alternative for non-responding patients to conventional management. It seems critical to elucidate the paracrine modulation of the immune system by MSC for their rational use in regenerative/immunoregulatory therapies.

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We describe the case of a patient with severe hemophilia A who underwent major orthopedic surgery managed postoperatively by telemedicine (TM). The case is a splendid example of the implementation of TM and good collaboration between a Comprehensive Hemophilia Treatment Center (CHTC) and a local hospital.

Introduction: The purpose of this study is to evaluate the degree of education and empowerment of the patients of the Haemophilia Unit at ‘La Paz’ University Hospital (Madrid, Spain). Materials and methods: Haemophilic patients attending routine haemophilia consultations were asked to complete a questionnaire with a view to determining the patient´s perceived knowledge about the disease; their ability to make individual and group decisions; and whether the frequency of their hospital visits could be reduced. Results: Patients were shown to have ample knowledge about the disease. Knowledge had been acquired chiefly through personal experience and from the interaction with healthcare providers. Nearly 70% of patients felt capable of making decisions on matters affecting their health. Over half of the patients considered that their hospital visits could be reduced if they had more information. Patients experiencing the largest number of bleeding episodes were those showing the highest levels of empowerment. Over half the patients were satisfied with the institutional support they received. Conclusion: These patients with haemophilia are in general reasonably empowered on account of their personal experiences and their interaction with the healthcare providers. Current widespread access to information makes it easier to step up educational interventions in patients at lower haemorrhagic risk.