Long-term follow-up of IPEX syndrome patients after different therapeutic strategies: An international multicenter retrospective study

Autor: Barzaghi, F., Hernandez, L.C.A., Neven, B., Ricci, S., Kucuk, Z.Y., Bleesing, J.J., Nademi, Z., Slatter, M.A., Ulloa, E.R., Shcherbina, A., Roppelt, A., Worth, A., Silva, J., Aiuti, A., Murguia-Favela, L., Speckmann, C., Carneiro-Sampaio, M., Fernandes, J.F., Baris, S., Ozen, A., Karakoc-Aydiner, E., Kiykim, A., Schulz, A., Steinmann, S., Notarangelo, L.D., Gambineri, E., Lionetti, P., Shearer, W.T., Forbes, L.R., Martinez, C., Moshous, D., Blanche, S., Fisher, A., Ruemmele, F.M., Tissandier, C., Ouachee-Chardin, M., Rieux-Laucat, F., Cavazzana, M., Qasim, W., Lucarelli, B., Albert, M.H., Kobayashi, I., Alonso, L., Heredia, C.D. de, Kanegane, H., Lawitschka, A., Seo, J.J., Gonzalez-Vicent, M., Diaz, M.A., Goyal, R.K., Sauer, M.G., Yesilipek, A., Kim, M., Yilmaz-Demirdag, Y., Bhatia, M., Khlevner, J., Padilla, E.J.R., Martino, S., Montin, D., Neth, O., Molinos-Quintana, A., Valverde-Fernandez, J., Broides, A., Pinsk, V., Ballauf, A., Haerynck, F., Bordon, V., Dhooge, C., Garcia-Lloret, M.L., Bredius, R.G., Kawak, K., Haddad, E., Seidel, M.G., Duckers, G., Pai, S.Y., Dvorak, C.C., Ehl, S., Locatelli, F., Goldman, F., Gennery, A.R., Cowan, M.J., Roncarolo, M.G., Bacchetta, R., PIDTC, IEWP, European Soc Blood Marrow

Publikováno v: The Journal of allergy and clinical immunology, vol 141, iss 3
Repositório Institucional da USP (Biblioteca Digital da Produção Intelectual)
Universidade de São Paulo (USP)
instacron:USP
JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY
Journal of Allergy and Clinical Immunology
Journal of Allergy and Clinical Immunology, 141(3), 1036
The Journal of allergy and clinical immunology
Repositorio Institucional de la Consejería de Sanidad de la Comunidad de Madrid
Consejería de Sanidad de la Comunidad de Madrid

Background Immunodysregulation polyendocrinopathy enteropathy x-linked(IPEX) syndromeis a monogenic autoimmune disease caused by FOXP3 mutations. Because it is a rare disease, the natural history and response to treatments, including allogeneic hemat

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::52a22a1221b397120fbc83bc4587d509
https://escholarship.org/uc/item/56g0d17d

Hematopoietic stem cell transplantation for advanced myelodysplastic syndrome in children: results of the EWOG-MDS 98 study

Autor: Strahm, B., Nollke, P., Zecca, M., Korthof, E.T., Bierings, M., Furlan, I., Sedlacek, P., Chybicka, A., Schmugge, M., Bordon, V., Peters, C., O'Marcaigh, A., Heredia, C.D. de, Bergstraesser, E., Moerloose, B.D., Heuvel-Eibrink, M.M. van den, Stary, J., Trebo, M., Wojcik, D., Niemeyer, C.M., Locatelli, F., EWOG-MDS Study Grp

Publikováno v: Leukemia, 25(3), 455-462
Leukemia, 25, 455-462. Nature Publishing Group

We report on the outcome of children with advanced primary myelodysplastic syndrome (MDS) transplanted from an HLA-matched sibling (MSD) or an unrelated donor (UD) following a preparative regimen with busulfan, cyclophosphamide and melphalan. Ninety-

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::db85d69872169fb987fc0f33fe0488f7
http://hdl.handle.net/1887/109076