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Your search keyword '"Cavazzana Calvo, M"' showing total 463 results
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463 results on '"Cavazzana Calvo, M"'

1. LMO2-Associated Clonal T Cell Proliferation in Two Patients after Gene Therapy for SCID-X1

Author

Von Kalle, C., Schmidt, M., McCormack, M. P., Wulffraat, N., Leboulch, P., Lim, A., Osborne, C. S., Pawliuk, R., Morillon, E., Sorensen, R., Forster, A., Fraser, P., Cohen, J. I., Alexander, I., Wintergerst, U., Frebourg, T., Aurias, A., Stoppa-Lyonnet, D., Romana, S., Radford-Weiss, I., Gross, F., Valensi, F., Delabesse, E., Macintyre, E., Sigaux, F., Soulier, J., Leiva, L. E., Wissler, M., Prinz, C., Rabbitts, T. H., Le Deist, F., Fischer, A., and Cavazzana-Calvo, M.

Published
2003

4. Severe Combined Immunodeficiencies in Humans

Author

de Villartay, J. P., de Saint Basile, G., Soudais, C., Le Deist, F., Hivroz, C., Rieux-Laucat, F., Cavazzana-Calvo, M., Disanto, J., Markievicz, S., Lisowska-Grospierre, B., Fischer, A., Gergely, János, editor, Benczúr, M., editor, Erdei, Anna, editor, Falus, A., editor, Füst, Gy., editor, Medgyesi, G., editor, Petrányi, Gy., editor, and Rajnavölgyi, Éva, editor

Published
1993
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14. LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1

Author

Hacein-Bey-Abina, S., Von Kalle, C., Schmidt, M., McCormack, M.P., Wulffraat, N., Leboulch, P., Lim, A., Osborne, C.S., Pawliuk, R., Morillon, E., Sorensen, R., Forster, A., Fraser, P., Cohen, J.I., de Saint Basile, G., Alexander, I., Wintergerst, U., Frebourg, T., Aurias, A., Stoppa-Lyonnet, D., Romana, S., Radford-Weiss, I., Gross, F., Valensi, F., Delabesse, E., Macintyre, E., Sigaux, F., Soulier, J., Leiva, L.E., Wissler, M., Prinz, C., Rabbitts, T.H., Le Deist, F., Fischer, A., and Cavazzana-Calvo, M.

Subjects
Physiological aspects, Complications and side effects, Research, Gene therapy -- Research -- Physiological aspects, Retroviruses -- Research -- Physiological aspects
Abstract

Ex vivo retrovirus-mediated gene transfer into hematopoietic progenitor cells has been shown to be an efficient strategy to correct inherited diseases of the lymphohematopoietic system, provided that a strong selective [...], We have previously shown correction of X-linked severe combined immunodeficiency [SCID-X1, also known as γ chain (γc) deficiency] in 9 out of 10 patients by retrovirus-mediated γc gene transfer into autologous CD34 bone marrow cells. However, almost 3 years after gene therapy, uncontrolled exponential clonal proliferation of mature T cells (with γδ+ or αβ+ T cell receptors) has occurred in the two youngest patients. Both patients' clones showed retrovirus vector integration in proximity to the LMO2 proto-oncogene promoter, leading to aberrant transcription and expression of LMO2. Thus, retrovirus vector insertion can trigger deregulated premalignant cell proliferation with unexpected frequency, most likely driven by retrovirus enhancer activity on the LMO2 gene promoter.

Published
2003

30. Where do we stand 6 years after gene therapy?

Author

Cavazzana-Calvo, M., Hacein-Bey-Abina, S., von Kalle, C., Schmidt, M., de Saint-Basile, G., Le Deist, F., Wulfraat, N., Alexander, I., Landais, P., Blanche, S., and Fischer, A.

Published
2005

36. Second allogeneic haematopoietic stem cell transplantation in relapsed acute and chronic leukaemias for patients who underwent a first allogeneic bone marrow transplantation: a survey of the Société Française de Greffe de Moelle (SFGM)

Author

Michallet, M., Tanguy, M. L., Socié, G., Thiébaut, A., Belhabri, A., Milpied, N., Reiffers, J., Kuentz, M., Cahn, J. Y., Blaise, D., Demeocq, F., Jouet, J. P., Michallet, A. S., Ifrah, N., Vilmer, E., Molina, L., Michel, G., Lioure, B., Cavazzana-Calvo, M., Pico, J. L., Sadoun, A., Guyotat, D., Attal, M., Curé, H., Bordigoni, P., Sutton, L., Buzyn-Veil, A., Tilly, M., Leporrier, M., Fegueux, N., Dreyfus, F., Rio, B., Lutz, P., and Vernant, J. P.

Published
2000

40. A phase II trial of partially incompatible bone marrow transplantation for high-risk acute lymphoblastic leukaemia in children: prevention of graft rejection with anti-LFA-1 and anti-CD2 antibodies

Author

CAVAZZANA-CALVO, M., BORDIGONI, P., MICHEL, G., ESPEROU, H., SOUILLET, G., LEBLANC, T., STEPHAN, J. L., VANNIER, J. P., MECHINAUD, F., REIFFERS, J., VILMER, E., LANDMAN-PARKER, J., BENKERROU, M., BARUCHEL, A., PICO, J., BERNAUDIN, F., BERGERON, C., PLOUVIER, E., THOMAS, C., WIJDENES, J., LACOUR, B., BLANCHE, S., and FISCHER, A.

Published
1996

45. LentiGlobin gene therapy for transfusion-dependent β-Thalassemia: Update from the northstar HGB-204 phase 1/2 clinical study

Author

Rasko, J., primary, Thompson, A., additional, Kwiatkowski, J., additional, Hongeng, S., additional, Schiller, G.J., additional, Anurathapan, U., additional, Cavazzana-Calvo, M., additional, Ho, P., additional, von Kalle, C., additional, Kletzel, M., additional, Leboulch, P., additional, Vichinsky, E., additional, Petrusich, A., additional, Asmal, M., additional, and Walters, M., additional

Published
2017
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46. Outcome of hematopoietic stem cell transplantation for adenosine deaminase-deficient severe combined immunodeficiency

Author

Hassan, A., Booth, C., Brightwell, A., Allwood, Z., Veys, P., Rao, K., Honig, M., Friedrich, W., Gennery, A., Slatter, M., Bredius, R., Finocchi, A., Cancrini, C., Aiuti, A., Porta, F., Lanfranchi, A., Ridella, M., Steward, C., Filipovich, A., Marsh, R., Bordon, V., Al-Muhsen, S., Al-Mousa, H., Alsum, Z., Al-Dhekri, H., Ghonaium, A. al, Speckmann, C., Fischer, A., Mahlaoui, N., Nichols, K.E., Grunebaum, E., Zahrani, D. al, Roifman, C.M., Boelens, J., Davies, E.G., Cavazzana-Calvo, M., Notarangelo, L., Gaspar, H.B., European Grp Blood Marrow Transpla, European Soc Immunodeficiency, Hassan, A, Booth, C, Brightwell, A, Allwood, Z, Veys, P, Rao, K, Hönig, M, Friedrich, W, Gennery, A, Slatter, M, Bredius, R, Finocchi, A, Cancrini, C, Aiuti, Alessandro, Porta, F, Lanfranchi, A, Ridella, M, Steward, C, Filipovich, A, Marsh, R, Bordon, V, AL MUHSEN, S, AL MOUSA, H, Alsum, Z, AL DHEKRI, H, AL GHONAIUM, A, Speckmann, C, Fischer, A, Mahlaoui, N, Nichols, Ke, Grunebaum, E, AL ZAHRANI, D, Roifman, Cm, Boelens, J, Davies, Eg, CAVAZZANA CALVO, M, Notarangelo, L, and Gaspar, Hb

Subjects
Oncology, Male, Transplantation Conditioning, Adenosine Deaminase, Genetic enhancement, medicine.medical_treatment, T-Lymphocytes, Hematopoietic stem cell transplantation, Kaplan-Meier Estimate, Biochemistry, humoral, Adenosine deaminase, newborn, immune system diseases, Agammaglobulinemia, hemic and lymphatic diseases, Child, Immunity, Cellular, Hematology, biology, Histocompatibility Testing, Graft Survival, Hematopoietic Stem Cell Transplantation, surgical procedures, operative, Treatment Outcome, Child, Preschool, Female, Unrelated Donors, medicine.medical_specialty, Immunology, preschool, Internal medicine, medicine, Humans, Lymphocyte Count, Retrospective Studies, Settore MED/38 - Pediatria Generale e Specialistica, Severe combined immunodeficiency, agammaglobulinemia, transplantation conditioning, severe combined immunodeficiency, humans, retrospective studies, infant, newborn, child, child, preschool, kaplan-meier estimate, infant, lymphocyte count, histocompatibility testing, immunity, cellular, unrelated donors, graft survival, treatment outcome, myeloablative agonists, hematopoietic stem cell transplantation, siblings, adenosine deaminase, male, female, t-lymphocytes, immunity, humoral, business.industry, Siblings, Infant, Newborn, Infant, Cell Biology, Myeloablative Agonists, medicine.disease, immunity, Adenosine deaminase deficiency, Immunity, Humoral, Transplantation, biology.protein, Severe Combined Immunodeficiency, business, cellular
Abstract

Deficiency of the purine salvage enzyme adenosine deaminase leads to SCID (ADA-SCID). Hematopoietic cell transplantation (HCT) can lead to a permanent cure of SCID; however, little data are available on outcome of HCT for ADA-SCID in particular. In this multicenter retrospective study, we analyzed outcome of HCT in 106 patients with ADA-SCID who received a total of 119 transplants. HCT from matched sibling and family donors (MSDs, MFDs) had significantly better overall survival (86% and 81%) in comparison with HCT from matched unrelated (66%; P < .05) and haploidentical donors (43%; P < .001). Superior overall survival was also seen in patients who received unconditioned transplants in comparison with myeloablative procedures (81% vs 54%; P < .003), although in unconditioned haploidentical donor HCT, nonengraftment was a major problem. Long-term immune recovery showed that regardless of transplant type, overall T-cell numbers were similar, although a faster rate of T-cell recovery was observed after MSD/MFD HCT. Humoral immunity and donor B-cell engraftment was achieved in nearly all evaluable surviving patients and was seen even after unconditioned HCT. These data detail for the first time the outcomes of HCT for ADA-SCID and show that, if patients survive HCT, long-term cellular and humoral immune recovery is achieved.

Published
2012

49. Transplantation in patients with SCID: mismatched related stem cells or unrelated cord blood?

Author

Fernandes, Jf, Rocha, V, Labopin, M, Neven, B, Moshous, D, Gennery, Ar, Friedrich, W, Porta, F, Diaz de Heredia, C, Wall, D, Bertrand, Y, Veys, P, Slatter, M, Schulz, A, Chan, Kw, Grimley, M, Ayas, M, Gungor, T, Ebell, W, Bonfim, C, Kalwak, K, Taupin, P, Blanche, S, Gaspar, Hb, Landais, P, Fischer, A, Gluckman, E, Cavazzana Calvo, M, Eurocord, Inborn Errors Working Party of European Group for Blood, Marrow Transplantation: Ahmed, A, Auiti, A, Biffi, A, Cant, A, Fasth, A, Gennery, A, Hassan, A, Lankester, A, O'Mera, A, Plabani, A, Rovelli, A, Salmon, A, Scarselli, A, Thrasher, A, Van Royen, A, Villa, A, Wawer, A, Wahadneh, A, Worth, A, Belohradsky, B, Wolska, B, Gaspar, B, Bonfirm, C, Booth, C, Klein, C, Messina, C, Peters, C, Steward, C, Lindemans, C, Schuetz, C, de Heredia Rubio CD, Bensoussan, D, Gleadow, D, Lilic, D, Gambineri, Eleonora, Smith, E, Aerts, F, Caracseghi, F, Roberts, G, Davies, G, Al Mousa, H, Jossanc, H, Ozsahim, H, Hirsch, I, Meyts, I, Tezcan, I, Mueller, I, Andresc, I, Boelens, J, Fernandes, J, Folloni, J, Keuhl, J, Reichenbach, J, Stary, J, Wachowiak, J, Xu Bayford, J, Cunha, Jm, Ehlert, J, Rao, K, Sykora, K, Andais, L, Brown, L, Dal Cortivo, L, Griffith, L, Notarangelo, L, Abinun, M, Albert, M, Bierings, M, Bouchet, M, Cavazzana, M, Hirschfield, M, Cowan, M, Hoenig, M, Loubser, M, Roncarolo, M, Sauer, M, Schneider, M, Verstegen, M, Schroeder, M, Essink, M, Yesilipek, M, Entz Werle, N, Mahlaoui, N, Schlautmann, N, Taylor, N, Vanroyen, N, Walffraat, N, Sanal, O, Amrolia, P, Bordigoni, P, De Coppi, P, Frange, P, Orchard, P, Sedlacek, P, Shaw, P, Stephensky, P, Bacchetta, R, Bredius, R, Formankova, R, Gale, R, Seger, R, Wynn, R, Corbacioglu, S, Ehl, S, Hacein Bey, S, Hambleton, S, Mohsen, S, Mueller, S, Pai, Sy, Espanol, T, Flood, T, Guengoer, T, Bordon, V, Ormoor, V, Pashano, V, Courteille, V, Czogala, W, Qasim, W, Camci, Y, and Nademi, Z.

Subjects
Male, medicine.medical_specialty, Transplantation Conditioning, medicine.medical_treatment, Immunology, Graft vs Host Disease, Kaplan-Meier Estimate, Cord Blood Stem Cell Transplantation, Hematopoietic stem cell transplantation, Biochemistry, Gastroenterology, SCID HSCT, Internal medicine, medicine, Humans, Proportional Hazards Models, Retrospective Studies, Preparative Regimen, Severe combined immunodeficiency, business.industry, Umbilical Cord Blood Transplantation, Incidence, Hematopoietic Stem Cell Transplantation, Infant, Newborn, Infant, Cell Biology, Hematology, medicine.disease, Surgery, Transplantation, Treatment Outcome, Child, Preschool, Histocompatibility, Cord blood, Female, Severe Combined Immunodeficiency, business
Abstract

Pediatric patients with SCID constitute medical emergencies. In the absence of an HLA-identical hematopoietic stem cell (HSC) donor, mismatched related-donor transplantation (MMRDT) or unrelated-donor umbilical cord blood transplantation (UCBT) are valuable treatment options. To help transplantation centers choose the best treatment option, we retrospectively compared outcomes after 175 MMRDTs and 74 UCBTs in patients with SCID or Omenn syndrome. Median follow-up time was 83 months and 58 months for UCBT and MMRDT, respectively. Most UCB recipients received a myeloablative conditioning regimen; most MMRDT recipients did not. UCB recipients presented a higher frequency of complete donor chimerism (P = .04) and faster total lymphocyte count recovery (P = .04) without any statistically significance with the preparative regimen they received. The MMRDT and UCBT groups did not differ in terms of T-cell engraftment, CD4+ and CD3+ cell recoveries, while Ig replacement therapy was discontinued sooner after UCBT (adjusted P = .02). There was a trend toward a greater incidence of grades II-IV acute GVHD (P = .06) and more chronic GVHD (P = .03) after UCBT. The estimated 5-year overall survival rates were 62% ± 4% after MMRDT and 57% ± 6% after UCBT. For children with SCID and no HLA-identical sibling donor, both UCBT and MMRDT represent available HSC sources for transplantation with quite similar outcomes.

Published
2012

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