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Recommendations for Improving the Quality of Rare Disease Registries
Kodra, Y.; Weinbach, J.; Posada-de-la-Paz, M.; Coi, A.; Lemonnier, S.L.; Enckevort, D. van; ... ; Taruscio, D. 2018
Recommendations for Improving the Quality of Rare Disease Registries
Article / Letter to editor
open access
Clinical presentation of patients suffering from recent onset chronic inflammatory back pain suggestive of spondyloarthritis: The DESIR cohort
Dougados, M.; Etcheto, A.; Molto, A.; Alonso, S.; Bouvet, S.; Daures, J.P.; ... ; Wendling, D. 2015
Clinical presentation of patients suffering from recent onset chronic inflammatory back pain suggestive of spondyloarthritis: The DESIR cohort
Article / Letter to editor
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Transplantation in patients with SCID: mismatched related stem cells or unrelated cord blood?
Fernandes, J.F.; Rocha, V.; Labopin, M.; Neven, B.; Moshous, D.; Gennery, A.R.; ... ; Eurocord Inborn Errors Working 2012
Transplantation in patients with SCID: mismatched related stem cells or unrelated cord blood?
Article / Letter to editor
metadata only
Transplantation of hematopoietic stem cells and long-term survival for primary immunodeficiencies in Europe: Entering a new century, do we do better?
Gennery, A.R.; Slatter, M.A.; Grandin, L.; Taupin, P.; Cant, A.J.; Veys, P.; ... ; European Society For 2010
Transplantation of hematopoietic stem cells and long-term survival for primary immunodeficiencies in Europe: Entering a new century, do we do better?
Article / Letter to editor
metadata only
Background: Hematopoietic stem cell transplantation remains the only treatment for most patients with severe combined immunodeficiencies (SCIDs) or other primary immunodeficiencies (non-SCID PIDs).... Show moreBackground: Hematopoietic stem cell transplantation remains the only treatment for most patients with severe combined immunodeficiencies (SCIDs) or other primary immunodeficiencies (non-SCID PIDs). Objective: To analyze the long-term outcome of patients with SCID and non-SCID PID from European centers treated between 1968 and 2005. Methods: The product-limit method estimated cumulative survival; the log-rank test compared survival between groups. A Cox proportional-hazard model evaluated the impact of independent predictors on patient survival. Results: In patients with SCID, survival with genoidentical donors (n = 25) from 2000 to 2005 was 90%. Survival using a mismatched relative (n = 96) has improved (66%), similar to that using an unrelated donor (n = 46; 69%; P = .005). Transplantation after year 1995, a younger age, B+ phenotype, genoidentical and phenoidentical donors, absence of respiratory impairment, or viral infection before transplantation were associated with better prognosis on multivariate analysis. For nonSCID PID, in contrast with patients with SCID, we confirm that, in the 2000 to 2005 period, using an unrelated donor (n = 124) gave a 3-year survival rate similar to a genoidentical donor (n = 73), 79% for both. Survival was 76% in phenoidentical transplants (n = 23) and worse in mismatched related donor transplants (n = 47; 46%; P = .016). Conclusion: This is the largest cohort study of such patients with the longest follow-up. Specific issues arise for different patient groups. Patients with B-SCID have worse survival than other patients with SCID, despite improvements in each group. For non-SCID PID, survival is worse than SCID, although more conditions are now treated. Individual disease categories now need to be analyzed so that disease-specific prognosis may be better understood and the best treatments planned. (J Allergy Clin Immunol 2010;126:602-10.) Show less