Orphan devices: yesterday is history; tomorrow is mystery: towards a European orphan device directive?

Abstract Background Regulatory and economic frameworks stimulated the research and development of orphan drugs, but very little has been done for devices necessary for the in-vivo diagnosis, prevention and treatment of life-threatening conditions with a low prevalence/incidence. Discussion A general public consultation in Europe has shown a positive attitude towards an “orphan device” directive. The United States of America have a Humanitarian Use Device exemption, but Europe is still waiting for such a stimulating framework. Post-marketing surveillance (“materio-vigilance”) will be necessary for follow-up, patient-reported outcome measures (quality of life versus survival) needed and off-label use data available for patient-safety reasons. Summary The marketing period for devices is shorter than for medicinal products. Incentives are necessary to stimulate research and development of such “orphan devices” especially when surgical intervention is the only option

Editorial: Prevention, diagnosis and treatment of rare disorders

Peer reviewed: Tru

High-dose growth hormone therapy for short children born small for gestational age

SCOPUS: ar.jFLWNAinfo:eu-repo/semantics/publishe

Patterns of Locoregional Relapses in Patients with Contemporarily Staged Stage III-N2 NSCLC Treated with Induction Chemotherapy and Resection: Implications for Postoperative Radiotherapy Target Volumes

Objectives: Our aim was to evaluate locoregional relapse (LR) patterns after induction chemotherapy and surgery for stage III-N2 NSCLC staged with current standard methods and their impact on radiation target volumes for postoperative radiotherapy (PORT). Methods: A total of 150 patients with stage III-N2 NSCLC from a prospective database of patients who underwent surgical resection at the University Hospitals of Leuven or the Oncologic Centre Limburg between 1998 and 2012 were included. Patients were staged with fluorodeoxyglucose F 18 positron emission tomography/computed tomography and brain imaging and treated with induction chemotherapy and surgery. PORT was performed for incomplete resection (R1/R2) and/or persistent nodal disease (ypN2). For the non-PORT group, we created a virtual planning target volume (PTV). In general, the clinical target volume encompassed the bronchial stump, the ipsilateral hilum, the subcarinal region (station 7), and the initially involved mediastinal lymph nodes. Results: After a mean follow-up time of 49 months, the 5-year overall survival was 35.1% in all patients; disease free survival was 31.8%. PORT was delivered to 70 patients. LR was seen in 26 patients in the PORT group (37%) and 32 in the non-PORT group (40%). Fifty-eight nodal relapse sites were seen in the PORT group (2.2 sites per patient) versus 113 in the non-PORT group (3.5 sites per patient) (p <0.01). In the PORT group, the most frequent sites of LR were the ipsilateral hilum (21%), lymph node station 7 (15%), ipsilateral station 4 (9%), ipsilateral station 5 (9%) and ipsilateral station 6 (9%). For the non PORT group these were station 7 (19%), ipsilateral 4 (16%), and ipsilateral hilum (14%). The dominant pattern of failure was inside (inside or both inside and outside) the PTV. Regarding the out-of-PTV relapses, 47% and 69% of LRs occurred in the contralateral mediastinum for the PORT and non-PORT groups, respectively. Out-of-PTV relapses occurred mostly in initially left-sided tumors. Conclusions: Despite the limitations of this retrospective study, our data support the role of PORT in decreasing local relapses. Because of the large number of out-of-PTV relapses in the contralateral mediastinum, inclusion of elective contralateral lymph node stations in the PTV could be considered in left-sided tumors. However, prospective randomized trials are needed to verify this

Final height in children with idiopathic growth hormone deficiency treated with recombinant human growth hormone: The Belgian experience

Background: The growth response to recombinant hGH (rhGH) treatment and final height of 61 Belgian children (32 boys) with idiopathic growth hormone deficiency (GHD) were studied. Patients/Methods: Two patient groups were compared: Group 1 with spontaneous puberty (n = 49), Group 2 with induced puberty (n = 12). The patients were treated with daily subcutaneous injections of rhGH in a dose of 0.5-0.7 IU/kg/week (0.17-0.23 mg/ kg/week) from the mean SD age of 11.9 +/- 3.1 years during 5.1 +/- 2.1 years. Results: rhGH treatment induced a doubling of the height velocity during the first year and resulted in a normalisation of height in 53 (87%) patients. Final height was -0.7 +/- 1.1 SDS, being 170.4 +/- 7.2 cm in boys and 158.0 +/- 6.4 cm in girls. Corrected for mid-parental height, final height was 0.0 +/- 1.1 SIDS. Ninety-two percent of the patients attained an adult height within the genetically determined target height range. Although height gain during puberty was smaller in the patients with induced puberty (boys: 17.1 +/- 7.0 cm vs. 27.5 +/- 6.6 cm (p < 0.005); girls: 9.6 +/- 7.4 cm vs. 22.2 +/- 6.1 cm (p < 0.005)), no differences in final height after adjustment for mid-parental height were found between patients with spontaneous or induced puberty. Conclusions: We conclude that patients with idiopathic GHD treated with rhGH administered as daily subcutaneous injections in a dose of 0.5-0.7 IU/kg/week reach their genetic growth potential, resulting in a normalisation of height in the majority of them, irrespective of spontaneous or induced puberty. Copyright (C) 2001 S. Karger AG, Basel

De quelques usages pédagogiques des sciences de l'éducation

Etévé Christiane, Binsse Thérèse, Brouaux Emmanuelle, Bourgeois Yvette, Douieb El Attafi Abdellatif, Dooms Chantal, Duchan Francis, Dupire Marc, Drumetz Jean-Claude, Hannebique Anne, Leroy Jean-Pierre, Macou Madeleine, Moreau Marc, Cognet Sylviane, Vanhecke-Dugez M., Lequeux Claudine, Denorme Eliane, Kurek Léo, Pouillard Catherine, Houdi Elgah Naziha, Hedoux Jacques. De quelques usages pédagogiques des sciences de l'éducation. In: Revue française de pédagogie, volume 79, 1987. pp. 105-111