-
1
-
-
0014433553
-
Immunological reconstitution of sex-linked lymphopenic immunological deficiency
-
Gatti R.A., Meuwissen H.J., Allen H.D., et al. Immunological reconstitution of sex-linked lymphopenic immunological deficiency. Lancet 1968, 2:1366-1369.
-
(1968)
Lancet
, vol.2
, pp. 1366-1369
-
-
Gatti, R.A.1
Meuwissen, H.J.2
Allen, H.D.3
-
2
-
-
0014433499
-
Bone-marrow transplantation in a patient with the Wiskott-Aldrich syndrome
-
Bach F.H., Albertini R.J., Joo P., et al. Bone-marrow transplantation in a patient with the Wiskott-Aldrich syndrome. Lancet 1968, 2:1364-1366.
-
(1968)
Lancet
, vol.2
, pp. 1364-1366
-
-
Bach, F.H.1
Albertini, R.J.2
Joo, P.3
-
3
-
-
57149131532
-
Allogeneic hematopoietic cell transplantation for primary immune deficiency diseases: current status and critical needs
-
Griffith L.M., Cowan M.J., Kohn D.B., et al. Allogeneic hematopoietic cell transplantation for primary immune deficiency diseases: current status and critical needs. JAllergy Clin Immunol 2008, 122:1087-1096.
-
(2008)
JAllergy Clin Immunol
, vol.122
, pp. 1087-1096
-
-
Griffith, L.M.1
Cowan, M.J.2
Kohn, D.B.3
-
4
-
-
84900846113
-
Primary immunodeficiency diseases: an update on the classification from the international union of immunological societies expert committee for primary immunodeficiency
-
Al-Herz W., Bousfiha A., Casanova J.L., et al. Primary immunodeficiency diseases: an update on the classification from the international union of immunological societies expert committee for primary immunodeficiency. Front Immunol 2014, 5:162.
-
(2014)
Front Immunol
, vol.5
, pp. 162
-
-
Al-Herz, W.1
Bousfiha, A.2
Casanova, J.L.3
-
5
-
-
2542461255
-
Molecular defects in human severe combined immunodeficiency and approaches to immune reconstitution
-
Buckley R.H. Molecular defects in human severe combined immunodeficiency and approaches to immune reconstitution. Annu Rev Immunol 2004, 22:625-655.
-
(2004)
Annu Rev Immunol
, vol.22
, pp. 625-655
-
-
Buckley, R.H.1
-
6
-
-
11244284216
-
Improved survival after unrelated donor bone marrow transplantation in children with primary immunodeficiency using a reduced-intensity conditioning regimen
-
Rao K., Amrolia P.J., Jones A., et al. Improved survival after unrelated donor bone marrow transplantation in children with primary immunodeficiency using a reduced-intensity conditioning regimen. Blood 2005, 105:879-885.
-
(2005)
Blood
, vol.105
, pp. 879-885
-
-
Rao, K.1
Amrolia, P.J.2
Jones, A.3
-
7
-
-
0035885937
-
Transplacentally acquired maternal T lymphocytes in severe combined immunodeficiency: a study of 121 patients
-
Muller S.M., Ege M., Pottharst A., et al. Transplacentally acquired maternal T lymphocytes in severe combined immunodeficiency: a study of 121 patients. Blood 2001, 98:1847-1851.
-
(2001)
Blood
, vol.98
, pp. 1847-1851
-
-
Muller, S.M.1
Ege, M.2
Pottharst, A.3
-
8
-
-
34547134958
-
Unusual clinical and immunologic manifestations of transplacentally acquired maternal T cells in severe combined immunodeficiency
-
Palmer K., Green T.D., Roberts J.L., et al. Unusual clinical and immunologic manifestations of transplacentally acquired maternal T cells in severe combined immunodeficiency. JAllergy Clin Immunol 2007, 120:423-428.
-
(2007)
JAllergy Clin Immunol
, vol.120
, pp. 423-428
-
-
Palmer, K.1
Green, T.D.2
Roberts, J.L.3
-
9
-
-
84979421333
-
Graft versus graft and graft versus host reactions after HLA-identical bone marrow transplantation in a patient with severe combined immunodeficiency with transplacentally acquired lymphoid chimerism
-
Friedman N.J., Shiff S.E., Ward F.E., et al. Graft versus graft and graft versus host reactions after HLA-identical bone marrow transplantation in a patient with severe combined immunodeficiency with transplacentally acquired lymphoid chimerism. Pediatr Allergy Immunol 1991, 2:111-116.
-
(1991)
Pediatr Allergy Immunol
, vol.2
, pp. 111-116
-
-
Friedman, N.J.1
Shiff, S.E.2
Ward, F.E.3
-
10
-
-
0023896050
-
Accelerated development of immunity following transplantation of maternal marrow stem cells into infants with severe combined immunodeficiency and transplacentally acquired lymphoid chimerism
-
Barrett M.J., Buckley R.H., Schiff S.E., et al. Accelerated development of immunity following transplantation of maternal marrow stem cells into infants with severe combined immunodeficiency and transplacentally acquired lymphoid chimerism. Clin Exp Immunol 1988, 72:118-123.
-
(1988)
Clin Exp Immunol
, vol.72
, pp. 118-123
-
-
Barrett, M.J.1
Buckley, R.H.2
Schiff, S.E.3
-
11
-
-
77956395504
-
Transplantation of hematopoietic stem cells and long-term survival for primary immunodeficiencies in Europe: entering a new century, do we do better?
-
e1-11
-
Gennery A.R., Slatter M.A., Grandin L., et al. Transplantation of hematopoietic stem cells and long-term survival for primary immunodeficiencies in Europe: entering a new century, do we do better?. JAllergy Clin Immunol 2010, 126:602-610.e1-11.
-
(2010)
JAllergy Clin Immunol
, vol.126
, pp. 602-610
-
-
Gennery, A.R.1
Slatter, M.A.2
Grandin, L.3
-
12
-
-
0033580206
-
Hematopoietic stem-cell transplantation for the treatment of severe combined immunodeficiency
-
Buckley R.H., Schiff S.E., Schiff R.I., et al. Hematopoietic stem-cell transplantation for the treatment of severe combined immunodeficiency. NEngl J Med 1999, 340:508-516.
-
(1999)
NEngl J Med
, vol.340
, pp. 508-516
-
-
Buckley, R.H.1
Schiff, S.E.2
Schiff, R.I.3
-
13
-
-
0023026618
-
Bone-marrow transplantation for immunodeficiencies and osteopetrosis: European survey, 1968-1985
-
Fischer A., Griscelli C., Friedrich W., et al. Bone-marrow transplantation for immunodeficiencies and osteopetrosis: European survey, 1968-1985. Lancet 1986, 2:1080-1084.
-
(1986)
Lancet
, vol.2
, pp. 1080-1084
-
-
Fischer, A.1
Griscelli, C.2
Friedrich, W.3
-
14
-
-
0036464666
-
Hematopoietic stem cell transplantation for severe combined immunodeficiency in the neonatal period leads to superior thymic output and improved survival
-
Myers L.A., Patel D.D., Puck J.M., et al. Hematopoietic stem cell transplantation for severe combined immunodeficiency in the neonatal period leads to superior thymic output and improved survival. Blood 2002, 99:872-878.
-
(2002)
Blood
, vol.99
, pp. 872-878
-
-
Myers, L.A.1
Patel, D.D.2
Puck, J.M.3
-
15
-
-
79953082197
-
Neonatal diagnosis of severe combined immunodeficiency leads to significantly improved survival outcome: the case for newborn screening
-
Brown L., Xu-Bayford J., Allwood Z., et al. Neonatal diagnosis of severe combined immunodeficiency leads to significantly improved survival outcome: the case for newborn screening. Blood 2011, 117:3243-3246.
-
(2011)
Blood
, vol.117
, pp. 3243-3246
-
-
Brown, L.1
Xu-Bayford, J.2
Allwood, Z.3
-
16
-
-
84904876386
-
Transplantation of 240 patients with severe combined immunodeficiency (SCID) from 2000-2009: a Primary Immune Deficiency Treatment Consortium report
-
Pai S.Y., Logan B.R., Griffith L.M., et al. Transplantation of 240 patients with severe combined immunodeficiency (SCID) from 2000-2009: a Primary Immune Deficiency Treatment Consortium report. N Engl J Med 2014, 371:434-446.
-
(2014)
N Engl J Med
, vol.371
, pp. 434-446
-
-
Pai, S.Y.1
Logan, B.R.2
Griffith, L.M.3
-
17
-
-
13444301418
-
Development of population-based newborn screening for severe combined immunodeficiency
-
Chan K., Puck J.M. Development of population-based newborn screening for severe combined immunodeficiency. JAllergy Clin Immunol 2005, 115:391-398.
-
(2005)
JAllergy Clin Immunol
, vol.115
, pp. 391-398
-
-
Chan, K.1
Puck, J.M.2
-
18
-
-
34948870078
-
Group population-based newborn screening for severe combined immunodeficiency: steps toward implementation
-
Puck J.M., SCID Newborn Screening Working Group Group population-based newborn screening for severe combined immunodeficiency: steps toward implementation. JAllergy Clin Immunol 2007, 120:760-768.
-
(2007)
JAllergy Clin Immunol
, vol.120
, pp. 760-768
-
-
Puck, J.M.1
-
19
-
-
84857757608
-
The long quest for neonatal screening for severe combined immunodeficiency
-
[quiz: 605-6]
-
Buckley R.H. The long quest for neonatal screening for severe combined immunodeficiency. JAllergy Clin Immunol 2012, 129:597-604. [quiz: 605-6].
-
(2012)
JAllergy Clin Immunol
, vol.129
, pp. 597-604
-
-
Buckley, R.H.1
-
20
-
-
0033595133
-
Treatment of severe combined immunodeficiency
-
Flake A.W., Zanjani E.D. Treatment of severe combined immunodeficiency. NEngl J Med 1999, 341:291-292.
-
(1999)
NEngl J Med
, vol.341
, pp. 291-292
-
-
Flake, A.W.1
Zanjani, E.D.2
-
21
-
-
0029848406
-
Treatment of X-linked severe combined immunodeficiency by in utero transplantation of paternal bone marrow
-
Flake A.W., Roncarolo M.G., Puck J.M., et al. Treatment of X-linked severe combined immunodeficiency by in utero transplantation of paternal bone marrow. NEngl J Med 1996, 335:1806-1810.
-
(1996)
NEngl J Med
, vol.335
, pp. 1806-1810
-
-
Flake, A.W.1
Roncarolo, M.G.2
Puck, J.M.3
-
22
-
-
16144362330
-
In-utero transplantation of parental CD34 haematopoietic progenitor cells in a patient with X-linked severe combined immunodeficiency (SCIDXI)
-
Wengler G.S., Lanfranchi A., Frusca T., et al. In-utero transplantation of parental CD34 haematopoietic progenitor cells in a patient with X-linked severe combined immunodeficiency (SCIDXI). Lancet 1996, 348:1484-1487.
-
(1996)
Lancet
, vol.348
, pp. 1484-1487
-
-
Wengler, G.S.1
Lanfranchi, A.2
Frusca, T.3
-
23
-
-
32044459512
-
Bone marrow transplantation for severe combined immune deficiency
-
Grunebaum E., Mazzolari E., Porta F., et al. Bone marrow transplantation for severe combined immune deficiency. JAMA 2006, 295:508-518.
-
(2006)
JAMA
, vol.295
, pp. 508-518
-
-
Grunebaum, E.1
Mazzolari, E.2
Porta, F.3
-
24
-
-
84858851280
-
Transplantation in patients with SCID: mismatched related stem cells or unrelated cord blood?
-
Fernandes J.F., Rocha V., Labopin M., et al. Transplantation in patients with SCID: mismatched related stem cells or unrelated cord blood?. Blood 2012, 119:2949-2955.
-
(2012)
Blood
, vol.119
, pp. 2949-2955
-
-
Fernandes, J.F.1
Rocha, V.2
Labopin, M.3
-
25
-
-
84912099156
-
Comparison of outcomes of hematopoietic stem cell transplantation without chemotherapy conditioning by using matched sibling and unrelated donors for treatment of severe combined immunodeficiency.
-
in press
-
Dvorak C, Hassan A, Slatter M, etal. Comparison of outcomes of hematopoietic stem cell transplantation without chemotherapy conditioning by using matched sibling and unrelated donors for treatment of severe combined immunodeficiency. J Allergy Clin Immunol, in press.
-
J Allergy Clin Immunol
-
-
Dvorak, C.1
Hassan, A.2
Slatter, M.3
-
26
-
-
84901804522
-
Absence of host natural killer cells is associated with engraftment permissiveness following non-conditioned allogeneic haematopoietic stem cell transplant for sever combined immunodeficiency
-
Hassan A., Lee P., Maggina P., et al. Absence of host natural killer cells is associated with engraftment permissiveness following non-conditioned allogeneic haematopoietic stem cell transplant for sever combined immunodeficiency. JAllergy Clin Immunol 2014, 133:1660-1666.
-
(2014)
JAllergy Clin Immunol
, vol.133
, pp. 1660-1666
-
-
Hassan, A.1
Lee, P.2
Maggina, P.3
-
27
-
-
0027477193
-
Immune reconstitution in severe combined immunodeficiency disease after lectin-treated, T-cell-depleted haplocompatible bone marrow transplantation
-
Dror Y., Gallagher R., Wara D.W., et al. Immune reconstitution in severe combined immunodeficiency disease after lectin-treated, T-cell-depleted haplocompatible bone marrow transplantation. Blood 1993, 81:2021-2030.
-
(1993)
Blood
, vol.81
, pp. 2021-2030
-
-
Dror, Y.1
Gallagher, R.2
Wara, D.W.3
-
28
-
-
3242757373
-
Characterization of human FCGR3B*02 (HNA-1b, NA2) cDNAs and IMGT standardized description of FCGR3B alleles
-
Bertrand G., Duprat E., Lefranc M.P., et al. Characterization of human FCGR3B*02 (HNA-1b, NA2) cDNAs and IMGT standardized description of FCGR3B alleles. Tissue Antigens 2004, 64:119-131.
-
(2004)
Tissue Antigens
, vol.64
, pp. 119-131
-
-
Bertrand, G.1
Duprat, E.2
Lefranc, M.P.3
-
29
-
-
3242747324
-
Regulation of thymus size by competition for stromal niches among early T cell progenitors
-
Prockop S.E., Petrie H.T. Regulation of thymus size by competition for stromal niches among early T cell progenitors. JImmunol 2004, 173:1604-1611.
-
(2004)
JImmunol
, vol.173
, pp. 1604-1611
-
-
Prockop, S.E.1
Petrie, H.T.2
-
30
-
-
39149139612
-
Long-term immune reconstitution after anti-CD52-treated or anti-CD34-treated hematopoietic stem cell transplantation for severe T-lymphocyte immunodeficiency
-
Slatter M.A., Brigham K., Dickinson A.M., et al. Long-term immune reconstitution after anti-CD52-treated or anti-CD34-treated hematopoietic stem cell transplantation for severe T-lymphocyte immunodeficiency. JAllergy Clin Immunol 2008, 121:361-367.
-
(2008)
JAllergy Clin Immunol
, vol.121
, pp. 361-367
-
-
Slatter, M.A.1
Brigham, K.2
Dickinson, A.M.3
-
31
-
-
33745954723
-
Early determinants of long-term T-cell reconstitution after hematopoietic stem cell transplantation for severe combined immunodeficiency
-
Borghans J.A., Bredius R.G., Hazenberg M.D., et al. Early determinants of long-term T-cell reconstitution after hematopoietic stem cell transplantation for severe combined immunodeficiency. Blood 2006, 108:763-769.
-
(2006)
Blood
, vol.108
, pp. 763-769
-
-
Borghans, J.A.1
Bredius, R.G.2
Hazenberg, M.D.3
-
32
-
-
34248390176
-
Long-term T-cell reconstitution after hematopoietic stem-cell transplantation in primary T-cell-immunodeficient patients is associated with myeloid chimerism and possibly the primary disease phenotype
-
Cavazzana-Calvo M., Carlier F., Le Deist F., et al. Long-term T-cell reconstitution after hematopoietic stem-cell transplantation in primary T-cell-immunodeficient patients is associated with myeloid chimerism and possibly the primary disease phenotype. Blood 2007, 109:4575-4581.
-
(2007)
Blood
, vol.109
, pp. 4575-4581
-
-
Cavazzana-Calvo, M.1
Carlier, F.2
Le Deist, F.3
-
33
-
-
84876417064
-
Haploidentical related-donor hematopoietic cell transplantation in children using megadoses of CliniMACs-selected CD34(+) cells and a fixed CD3(+) dose
-
Dvorak C.C., Gilman A.L., Horn B., et al. Haploidentical related-donor hematopoietic cell transplantation in children using megadoses of CliniMACs-selected CD34(+) cells and a fixed CD3(+) dose. Bone Marrow Transplant 2013, 48:508-513.
-
(2013)
Bone Marrow Transplant
, vol.48
, pp. 508-513
-
-
Dvorak, C.C.1
Gilman, A.L.2
Horn, B.3
-
34
-
-
35348869770
-
Long-term follow-up in patients with severe combined immunodeficiency treated by bone marrow transplantation
-
Friedrich W., Honig M., Muller S.M. Long-term follow-up in patients with severe combined immunodeficiency treated by bone marrow transplantation. Immunol Res 2007, 38:165-173.
-
(2007)
Immunol Res
, vol.38
, pp. 165-173
-
-
Friedrich, W.1
Honig, M.2
Muller, S.M.3
-
35
-
-
18244411938
-
Long-term immune reconstitution and outcome after HLA-nonidentical T-cell-depleted bone marrow transplantation for severe combined immunodeficiency: a European retrospective study of 116 patients
-
Haddad E., Landais P., Friedrich W., et al. Long-term immune reconstitution and outcome after HLA-nonidentical T-cell-depleted bone marrow transplantation for severe combined immunodeficiency: a European retrospective study of 116 patients. Blood 1998, 91:3646-3653.
-
(1998)
Blood
, vol.91
, pp. 3646-3653
-
-
Haddad, E.1
Landais, P.2
Friedrich, W.3
-
36
-
-
35148815335
-
Long-term immune reconstitution and clinical outcome after stem cell transplantation for severe T-cell immunodeficiency
-
Mazzolari E., Forino C., Guerci S., et al. Long-term immune reconstitution and clinical outcome after stem cell transplantation for severe T-cell immunodeficiency. JAllergy Clin Immunol 2007, 120:892-899.
-
(2007)
JAllergy Clin Immunol
, vol.120
, pp. 892-899
-
-
Mazzolari, E.1
Forino, C.2
Guerci, S.3
-
37
-
-
84881371331
-
Long-term outcome of non-ablative booster BMT in patients with SCID
-
Teigland C.L., Parrott R.E., Buckley R.H. Long-term outcome of non-ablative booster BMT in patients with SCID. Bone Marrow Transplant 2013, 48:1050-1055.
-
(2013)
Bone Marrow Transplant
, vol.48
, pp. 1050-1055
-
-
Teigland, C.L.1
Parrott, R.E.2
Buckley, R.H.3
-
38
-
-
84255197284
-
IL-21 is the primary common gamma chain-binding cytokine required for human B-cell differentiation invivo
-
Recher M., Berglund L.J., Avery D.T., et al. IL-21 is the primary common gamma chain-binding cytokine required for human B-cell differentiation invivo. Blood 2011, 118:6824-6835.
-
(2011)
Blood
, vol.118
, pp. 6824-6835
-
-
Recher, M.1
Berglund, L.J.2
Avery, D.T.3
-
39
-
-
84875740332
-
Post-transplantation B cell function in different molecular types of SCID
-
Buckley R.H., Win C.M., Moser B.K., et al. Post-transplantation B cell function in different molecular types of SCID. JClin Immunol 2013, 33:96-110.
-
(2013)
JClin Immunol
, vol.33
, pp. 96-110
-
-
Buckley, R.H.1
Win, C.M.2
Moser, B.K.3
-
40
-
-
84861482319
-
Non-infectious lung disease in patients with adenosine deaminase deficient severe combined immunodeficiency
-
Booth C., Algar V.E., Xu-Bayford J., et al. Non-infectious lung disease in patients with adenosine deaminase deficient severe combined immunodeficiency. JClin Immunol 2012, 32:449-453.
-
(2012)
JClin Immunol
, vol.32
, pp. 449-453
-
-
Booth, C.1
Algar, V.E.2
Xu-Bayford, J.3
-
41
-
-
84861703206
-
Pulmonary alveolar proteinosis in patients with adenosine deaminase deficiency
-
Grunebaum E., Cutz E., Roifman C.M. Pulmonary alveolar proteinosis in patients with adenosine deaminase deficiency. JAllergy Clin Immunol 2012, 129:1588-1593.
-
(2012)
JAllergy Clin Immunol
, vol.129
, pp. 1588-1593
-
-
Grunebaum, E.1
Cutz, E.2
Roifman, C.M.3
-
42
-
-
84867745449
-
Outcome of hematopoietic stem cell transplantation for adenosine deaminase deficient severe combined immunodeficiency
-
Hassan A., Booth C., Brightwell A., et al. Outcome of hematopoietic stem cell transplantation for adenosine deaminase deficient severe combined immunodeficiency. Blood 2012, 120:3615-3624.
-
(2012)
Blood
, vol.120
, pp. 3615-3624
-
-
Hassan, A.1
Booth, C.2
Brightwell, A.3
-
43
-
-
84892636089
-
SCID patients with ARTEMIS vs RAG deficiencies following HCT: increased risk of late toxicity in ARTEMIS-deficient SCID
-
Schuetz C., Neven B., Dvorak C.C., et al. SCID patients with ARTEMIS vs RAG deficiencies following HCT: increased risk of late toxicity in ARTEMIS-deficient SCID. Blood 2014, 123:281-289.
-
(2014)
Blood
, vol.123
, pp. 281-289
-
-
Schuetz, C.1
Neven, B.2
Dvorak, C.C.3
-
44
-
-
0035042875
-
Bone marrow transplantation for T-B- severe combined immunodeficiency disease in Athabascan-speaking native Americans
-
O'Marcaigh A.S., DeSantes K., Hu D., et al. Bone marrow transplantation for T-B- severe combined immunodeficiency disease in Athabascan-speaking native Americans. Bone Marrow Transplant 2001, 27:703-709.
-
(2001)
Bone Marrow Transplant
, vol.27
, pp. 703-709
-
-
O'Marcaigh, A.S.1
DeSantes, K.2
Hu, D.3
-
45
-
-
84876188593
-
Multiple osteochondromas following irradiation-containing conditioning in severe combined immunodeficiency
-
Grunebaum E., Daneman A., Murguia-Favela L., et al. Multiple osteochondromas following irradiation-containing conditioning in severe combined immunodeficiency. Br J Haematol 2013, 161:446-448.
-
(2013)
Br J Haematol
, vol.161
, pp. 446-448
-
-
Grunebaum, E.1
Daneman, A.2
Murguia-Favela, L.3
-
46
-
-
69949094169
-
Haemopoietic stem-cell transplantation with antibody-based minimal-intensity conditioning: a phase 1/2 study
-
Straathof K.C., Rao K., Eyrich M., et al. Haemopoietic stem-cell transplantation with antibody-based minimal-intensity conditioning: a phase 1/2 study. Lancet 2009, 374:912-920.
-
(2009)
Lancet
, vol.374
, pp. 912-920
-
-
Straathof, K.C.1
Rao, K.2
Eyrich, M.3
-
47
-
-
79955951618
-
Radioimmunotherapy-based conditioning for hematopoietic cell transplantation in children with malignant and nonmalignant diseases
-
Schulz A.S., Glatting G., Hoenig M., et al. Radioimmunotherapy-based conditioning for hematopoietic cell transplantation in children with malignant and nonmalignant diseases. Blood 2011, 117:4642-4650.
-
(2011)
Blood
, vol.117
, pp. 4642-4650
-
-
Schulz, A.S.1
Glatting, G.2
Hoenig, M.3
-
48
-
-
55549090680
-
Reduced intensity HLA-haploidentical BMT with post transplantation cyclophosphamide in nonmalignant hematologic diseases
-
Brodsky R.A., Luznik L., Bolanos-Meade J., et al. Reduced intensity HLA-haploidentical BMT with post transplantation cyclophosphamide in nonmalignant hematologic diseases. Bone Marrow Transplant 2008, 42:523-527.
-
(2008)
Bone Marrow Transplant
, vol.42
, pp. 523-527
-
-
Brodsky, R.A.1
Luznik, L.2
Bolanos-Meade, J.3
-
49
-
-
84905095192
-
HLA-haploidentical stem cell transplantation after removal of alphabeta+ T and B-cells in children with non-malignant disorders
-
Bertaina A., Merli P., Rutella S., et al. HLA-haploidentical stem cell transplantation after removal of alphabeta+ T and B-cells in children with non-malignant disorders. Blood 2014, 124:822-826.
-
(2014)
Blood
, vol.124
, pp. 822-826
-
-
Bertaina, A.1
Merli, P.2
Rutella, S.3
-
50
-
-
33749247595
-
Outcome in patients with Wiskott-Aldrich syndrome following stem cell transplantation: an analysis of 57 patients in Japan
-
Kobayashi R., Ariga T., Nonoyama S., et al. Outcome in patients with Wiskott-Aldrich syndrome following stem cell transplantation: an analysis of 57 patients in Japan. Br J Haematol 2006, 135:362-366.
-
(2006)
Br J Haematol
, vol.135
, pp. 362-366
-
-
Kobayashi, R.1
Ariga, T.2
Nonoyama, S.3
-
51
-
-
33750546027
-
Stem cell transplantation for the Wiskott-Aldrich syndrome: a single-center experience confirms efficacy of matched unrelated donor transplantation
-
Pai S.Y., DeMartiis D., Forino C., et al. Stem cell transplantation for the Wiskott-Aldrich syndrome: a single-center experience confirms efficacy of matched unrelated donor transplantation. Bone Marrow Transplant 2006, 38:671-679.
-
(2006)
Bone Marrow Transplant
, vol.38
, pp. 671-679
-
-
Pai, S.Y.1
DeMartiis, D.2
Forino, C.3
-
52
-
-
69849087668
-
Results and long-term outcome in 39 patients with Wiskott-Aldrich syndrome transplanted from HLA-matched and -mismatched donors
-
Friedrich W., Schutz C., Schulz A., et al. Results and long-term outcome in 39 patients with Wiskott-Aldrich syndrome transplanted from HLA-matched and -mismatched donors. Immunol Res 2009, 44:18-24.
-
(2009)
Immunol Res
, vol.44
, pp. 18-24
-
-
Friedrich, W.1
Schutz, C.2
Schulz, A.3
-
53
-
-
80051641437
-
Long-term outcome and lineage-specific chimerism in 194 patients with Wiskott-Aldrich syndrome treated by hematopoietic cell transplantation in the period 1980-2009: an international collaborative study
-
Moratto D., Giliani S., Bonfim C., et al. Long-term outcome and lineage-specific chimerism in 194 patients with Wiskott-Aldrich syndrome treated by hematopoietic cell transplantation in the period 1980-2009: an international collaborative study. Blood 2011, 118:1675-1684.
-
(2011)
Blood
, vol.118
, pp. 1675-1684
-
-
Moratto, D.1
Giliani, S.2
Bonfim, C.3
-
54
-
-
0035869537
-
Impact of donor type on outcome of bone marrow transplantation for Wiskott-Aldrich syndrome: collaborative study of the International Bone Marrow Transplant Registry and the National Marrow Donor Program
-
Filipovich A.H., Stone J.V., Tomany S.C., et al. Impact of donor type on outcome of bone marrow transplantation for Wiskott-Aldrich syndrome: collaborative study of the International Bone Marrow Transplant Registry and the National Marrow Donor Program. Blood 2001, 97:1598-1603.
-
(2001)
Blood
, vol.97
, pp. 1598-1603
-
-
Filipovich, A.H.1
Stone, J.V.2
Tomany, S.C.3
-
55
-
-
64749097074
-
Wiskott-Aldrich syndrome: diagnosis, clinical and laboratory manifestations, and treatment
-
Ochs H.D., Filipovich A.H., Veys P., et al. Wiskott-Aldrich syndrome: diagnosis, clinical and laboratory manifestations, and treatment. Biol Blood Marrow Transplant 2009, 15:84-90.
-
(2009)
Biol Blood Marrow Transplant
, vol.15
, pp. 84-90
-
-
Ochs, H.D.1
Filipovich, A.H.2
Veys, P.3
-
56
-
-
9144225345
-
Treatment of CD40 ligand deficiency by hematopoietic stem cell transplantation: a survey of the European experience, 1993-2002
-
Gennery A.R., Khawaja K., Veys P., et al. Treatment of CD40 ligand deficiency by hematopoietic stem cell transplantation: a survey of the European experience, 1993-2002. Blood 2004, 103:1152-1157.
-
(2004)
Blood
, vol.103
, pp. 1152-1157
-
-
Gennery, A.R.1
Khawaja, K.2
Veys, P.3
-
57
-
-
33750558068
-
Nonmyeloablative stem cell transplantation for nonmalignant diseases in children with severe organ dysfunction
-
Kikuta A., Ito M., Mochizuki K., et al. Nonmyeloablative stem cell transplantation for nonmalignant diseases in children with severe organ dysfunction. Bone Marrow Transplant 2006, 38:665-669.
-
(2006)
Bone Marrow Transplant
, vol.38
, pp. 665-669
-
-
Kikuta, A.1
Ito, M.2
Mochizuki, K.3
-
58
-
-
0037394102
-
Polymerase chain reaction-based diagnosis of infection with Cryptosporidium in children with primary immunodeficiencies
-
McLauchlin J., Amar C.F., Pedraza-Diaz S., et al. Polymerase chain reaction-based diagnosis of infection with Cryptosporidium in children with primary immunodeficiencies. Pediatr Infect Dis J 2003, 22:329-335.
-
(2003)
Pediatr Infect Dis J
, vol.22
, pp. 329-335
-
-
McLauchlin, J.1
Amar, C.F.2
Pedraza-Diaz, S.3
-
59
-
-
10644266286
-
Correction of the hyper-IgM syndrome after liver and bone marrow transplantation
-
Hadzic N., Pagliuca A., Rela M., et al. Correction of the hyper-IgM syndrome after liver and bone marrow transplantation. NEngl J Med 2000, 342:320-324.
-
(2000)
NEngl J Med
, vol.342
, pp. 320-324
-
-
Hadzic, N.1
Pagliuca, A.2
Rela, M.3
-
60
-
-
84872787816
-
Successful haploidentical PBSCT with subsequent T-cell addbacks in a boy with HyperIgM syndrome presenting as severe congenital neutropenia
-
Jasinska A., Kalwak K., Trelinska J., et al. Successful haploidentical PBSCT with subsequent T-cell addbacks in a boy with HyperIgM syndrome presenting as severe congenital neutropenia. Pediatr Transplant 2013, 17:E37-40.
-
(2013)
Pediatr Transplant
, vol.17
, pp. E37-40
-
-
Jasinska, A.1
Kalwak, K.2
Trelinska, J.3
-
61
-
-
79957875084
-
Chronic granulomatous disease: overview and hematopoietic stem cell transplantation
-
[quiz: 1327-8]
-
Kang E.M., Marciano B.E., DeRavin S., et al. Chronic granulomatous disease: overview and hematopoietic stem cell transplantation. JAllergy Clin Immunol 2011, 127:1319-1326. [quiz: 1327-8].
-
(2011)
JAllergy Clin Immunol
, vol.127
, pp. 1319-1326
-
-
Kang, E.M.1
Marciano, B.E.2
DeRavin, S.3
-
62
-
-
0037114622
-
Treatment of chronic granulomatous disease with myeloablative conditioning and an unmodified hemopoietic allograft: a survey of the European experience, 1985-2000
-
Seger R.A., Gungor T., Belohradsky B.H., et al. Treatment of chronic granulomatous disease with myeloablative conditioning and an unmodified hemopoietic allograft: a survey of the European experience, 1985-2000. Blood 2002, 100:4344-4350.
-
(2002)
Blood
, vol.100
, pp. 4344-4350
-
-
Seger, R.A.1
Gungor, T.2
Belohradsky, B.H.3
-
63
-
-
0035932520
-
Treatment of chronic granulomatous disease with nonmyeloablative conditioning and a T-cell-depleted hematopoietic allograft
-
Horwitz M.E., Barrett A.J., Brown M.R., et al. Treatment of chronic granulomatous disease with nonmyeloablative conditioning and a T-cell-depleted hematopoietic allograft. NEngl J Med 2001, 344:881-888.
-
(2001)
NEngl J Med
, vol.344
, pp. 881-888
-
-
Horwitz, M.E.1
Barrett, A.J.2
Brown, M.R.3
-
64
-
-
84895068883
-
Reduced-intensity conditioning and HLA-matched haemopoietic stem-cell transplantation in patients with chronic granulomatous disease: a prospective multicentre study
-
Gungor T., Teira P., Slatter M., et al. Reduced-intensity conditioning and HLA-matched haemopoietic stem-cell transplantation in patients with chronic granulomatous disease: a prospective multicentre study. Lancet 2014, 383:436-448.
-
(2014)
Lancet
, vol.383
, pp. 436-448
-
-
Gungor, T.1
Teira, P.2
Slatter, M.3
-
65
-
-
64149091997
-
Acase of McLeod phenotype chronic granulomatous disease who received unrelated cord blood transplantation
-
Nakano T., Boku E., Yoshioka A., et al. Acase of McLeod phenotype chronic granulomatous disease who received unrelated cord blood transplantation. JPediatr Hematol Oncol 1999, 12:264.
-
(1999)
JPediatr Hematol Oncol
, vol.12
, pp. 264
-
-
Nakano, T.1
Boku, E.2
Yoshioka, A.3
-
66
-
-
84876451718
-
Health related quality of life and emotional health in children with chronic granulomatous disease: a comparison of those managed conservatively with those that have undergone haematopoietic stem cell transplant
-
Cole T., McKendrick F., Titman P., et al. Health related quality of life and emotional health in children with chronic granulomatous disease: a comparison of those managed conservatively with those that have undergone haematopoietic stem cell transplant. JClin Immunol 2013, 33:8-13.
-
(2013)
JClin Immunol
, vol.33
, pp. 8-13
-
-
Cole, T.1
McKendrick, F.2
Titman, P.3
-
67
-
-
78650918337
-
Residual NADPH oxidase and survival in chronic granulomatous disease
-
Kuhns D.B., Alvord W.G., Heller T., et al. Residual NADPH oxidase and survival in chronic granulomatous disease. NEngl J Med 2010, 363:2600-2610.
-
(2010)
NEngl J Med
, vol.363
, pp. 2600-2610
-
-
Kuhns, D.B.1
Alvord, W.G.2
Heller, T.3
-
68
-
-
36048955162
-
HLA-matched related donor hematopoietic cell transplantation in 43 patients with Fanconi anemia conditioned with 60 mg/kg of cyclophosphamide
-
Bonfim C.M., de Medeiros C.R., Bitencourt M.A., et al. HLA-matched related donor hematopoietic cell transplantation in 43 patients with Fanconi anemia conditioned with 60 mg/kg of cyclophosphamide. Biol Blood Marrow Transplant 2007, 13:1455-1460.
-
(2007)
Biol Blood Marrow Transplant
, vol.13
, pp. 1455-1460
-
-
Bonfim, C.M.1
de Medeiros, C.R.2
Bitencourt, M.A.3
-
69
-
-
51649094272
-
HLA-matched sibling hematopoietic stem cell transplantation for Fanconi anemia: comparison of irradiation and nonirradiation containing conditioning regimens
-
Pasquini R., Carreras J., Pasquini M.C., et al. HLA-matched sibling hematopoietic stem cell transplantation for Fanconi anemia: comparison of irradiation and nonirradiation containing conditioning regimens. Biol Blood Marrow Transplant 2008, 14:1141-1147.
-
(2008)
Biol Blood Marrow Transplant
, vol.14
, pp. 1141-1147
-
-
Pasquini, R.1
Carreras, J.2
Pasquini, M.C.3
-
71
-
-
34247842848
-
Successful bone marrow transplantation in a patient with DNA ligase IV deficiency and bone marrow failure
-
Gruhn B., Seidel J., Zintl F., et al. Successful bone marrow transplantation in a patient with DNA ligase IV deficiency and bone marrow failure. Orphanet J Rare Dis 2007, 2:5.
-
(2007)
Orphanet J Rare Dis
, vol.2
, pp. 5
-
-
Gruhn, B.1
Seidel, J.2
Zintl, F.3
-
72
-
-
30944455282
-
Severe combined immunodeficiency and microcephaly in siblings with hypomorphic mutations in DNA ligase IV
-
Buck D., Moshous D., de Chasseval R., et al. Severe combined immunodeficiency and microcephaly in siblings with hypomorphic mutations in DNA ligase IV. Eur J Immunol 2006, 36:224-235.
-
(2006)
Eur J Immunol
, vol.36
, pp. 224-235
-
-
Buck, D.1
Moshous, D.2
de Chasseval, R.3
-
73
-
-
84863727700
-
Two SCID cases with cernunnos-XLF deficiency successfully treated by hematopoietic stem cell transplantation
-
Cagdas D., Ozgur T.T., Asal G.T., et al. Two SCID cases with cernunnos-XLF deficiency successfully treated by hematopoietic stem cell transplantation. Pediatr Transplant 2012, 16:E167-171.
-
(2012)
Pediatr Transplant
, vol.16
, pp. E167-171
-
-
Cagdas, D.1
Ozgur, T.T.2
Asal, G.T.3
-
74
-
-
68849087197
-
Unrelated hematopoietic stem cell transplantation for Cernunnos-XLF deficiency
-
Faraci M., Lanino E., Micalizzi C., et al. Unrelated hematopoietic stem cell transplantation for Cernunnos-XLF deficiency. Pediatr Transplant 2009, 13:785-789.
-
(2009)
Pediatr Transplant
, vol.13
, pp. 785-789
-
-
Faraci, M.1
Lanino, E.2
Micalizzi, C.3
-
75
-
-
84880448795
-
Outcomes of allogeneic hematopoietic cell transplantation in patients with dyskeratosis congenita
-
Gadalla S.M., Sales-Bonfim C., Carreras J., et al. Outcomes of allogeneic hematopoietic cell transplantation in patients with dyskeratosis congenita. Biol Blood Marrow Transplant 2013, 19:1238-1243.
-
(2013)
Biol Blood Marrow Transplant
, vol.19
, pp. 1238-1243
-
-
Gadalla, S.M.1
Sales-Bonfim, C.2
Carreras, J.3
-
76
-
-
84883742527
-
Reduced intensity conditioning is effective for hematopoietic SCT in dyskeratosis congenita-related BM failure
-
Ayas M., Nassar A., Hamidieh A.A., et al. Reduced intensity conditioning is effective for hematopoietic SCT in dyskeratosis congenita-related BM failure. Bone Marrow Transplant 2013, 48:1168-1172.
-
(2013)
Bone Marrow Transplant
, vol.48
, pp. 1168-1172
-
-
Ayas, M.1
Nassar, A.2
Hamidieh, A.A.3
-
77
-
-
33845619137
-
HLH-2004: Diagnostic and therapeutic guidelines for hemophagocytic lymphohistiocytosis
-
Henter J.I., Horne A., Arico M., et al. HLH-2004: Diagnostic and therapeutic guidelines for hemophagocytic lymphohistiocytosis. Pediatr Blood Cancer 2007, 48:124-131.
-
(2007)
Pediatr Blood Cancer
, vol.48
, pp. 124-131
-
-
Henter, J.I.1
Horne, A.2
Arico, M.3
-
78
-
-
0027496475
-
Treatment of familial hemophagocytic lymphohistiocytosis with antithymocyte globulins, steroids, and cyclosporin A
-
Stephan J.L., Donadieu J., Ledeist F., et al. Treatment of familial hemophagocytic lymphohistiocytosis with antithymocyte globulins, steroids, and cyclosporin A. Blood 1993, 82:2319-2323.
-
(1993)
Blood
, vol.82
, pp. 2319-2323
-
-
Stephan, J.L.1
Donadieu, J.2
Ledeist, F.3
-
79
-
-
33646346117
-
Hematopoietic stem cell transplantation in hemophagocytic lymphohistiocytosis: a single-center report of 48 patients
-
Ouachee-Chardin M., Elie C., de Saint Basile G., et al. Hematopoietic stem cell transplantation in hemophagocytic lymphohistiocytosis: a single-center report of 48 patients. Pediatrics 2006, 117:e743-750.
-
(2006)
Pediatrics
, vol.117
, pp. e743-750
-
-
Ouachee-Chardin, M.1
Elie, C.2
de Saint Basile, G.3
-
80
-
-
78650637431
-
Reduced-intensity conditioning significantly improves survival of patients with hemophagocytic lymphohistiocytosis undergoing allogeneic hematopoietic cell transplantation
-
Marsh R.A., Vaughn G., Kim M.O., et al. Reduced-intensity conditioning significantly improves survival of patients with hemophagocytic lymphohistiocytosis undergoing allogeneic hematopoietic cell transplantation. Blood 2010, 116:5824-5831.
-
(2010)
Blood
, vol.116
, pp. 5824-5831
-
-
Marsh, R.A.1
Vaughn, G.2
Kim, M.O.3
-
81
-
-
78650984696
-
X-linked lymphoproliferative disease due to SAP/SH2D1A deficiency: a multicenter study on the manifestations, management and outcome of the disease
-
Booth C., Gilmour K.C., Veys P., et al. X-linked lymphoproliferative disease due to SAP/SH2D1A deficiency: a multicenter study on the manifestations, management and outcome of the disease. Blood 2011, 117:53-62.
-
(2011)
Blood
, vol.117
, pp. 53-62
-
-
Booth, C.1
Gilmour, K.C.2
Veys, P.3
-
82
-
-
84873606153
-
Allogeneic hematopoietic cell transplantation for XIAP deficiency: an international survey reveals poor outcomes
-
Marsh R.A., Rao K., Satwani P., et al. Allogeneic hematopoietic cell transplantation for XIAP deficiency: an international survey reveals poor outcomes. Blood 2013, 121:877-883.
-
(2013)
Blood
, vol.121
, pp. 877-883
-
-
Marsh, R.A.1
Rao, K.2
Satwani, P.3
-
83
-
-
67651056216
-
Hematopoietic stem cell transplantation in Griscelli syndrome type 2: a single-center report on 10 patients
-
Pachlopnik Schmid J., Moshous D., Boddaert N., et al. Hematopoietic stem cell transplantation in Griscelli syndrome type 2: a single-center report on 10 patients. Blood 2009, 114:211-218.
-
(2009)
Blood
, vol.114
, pp. 211-218
-
-
Pachlopnik Schmid, J.1
Moshous, D.2
Boddaert, N.3
-
84
-
-
22044433268
-
Progressive neurologic dysfunctions 20 years after allogeneic bone marrow transplantation for Chediak-Higashi syndrome
-
Tardieu M., Lacroix C., Neven B., et al. Progressive neurologic dysfunctions 20 years after allogeneic bone marrow transplantation for Chediak-Higashi syndrome. Blood 2005, 106:40-42.
-
(2005)
Blood
, vol.106
, pp. 40-42
-
-
Tardieu, M.1
Lacroix, C.2
Neven, B.3
-
85
-
-
84867742980
-
Immune dysregulation, polyendocrinopathy, enteropathy, x-linked syndrome: a paradigm of immunodeficiency with autoimmunity
-
Barzaghi F., Passerini L., Bacchetta R. Immune dysregulation, polyendocrinopathy, enteropathy, x-linked syndrome: a paradigm of immunodeficiency with autoimmunity. Front Immunol 2012, 3:211.
-
(2012)
Front Immunol
, vol.3
, pp. 211
-
-
Barzaghi, F.1
Passerini, L.2
Bacchetta, R.3
-
86
-
-
33845983223
-
Successful bone marrow transplantation for IPEX syndrome after reduced-intensity conditioning
-
Rao A., Kamani N., Filipovich A., et al. Successful bone marrow transplantation for IPEX syndrome after reduced-intensity conditioning. Blood 2007, 109:383-385.
-
(2007)
Blood
, vol.109
, pp. 383-385
-
-
Rao, A.1
Kamani, N.2
Filipovich, A.3
-
87
-
-
84893739675
-
Single centre experience of haematopoietic SCT for patients with immunodysregulation, polyendocrinopathy, enteropathy, X-linked syndrome
-
Nademi Z., Slatter M., Gambineri E., et al. Single centre experience of haematopoietic SCT for patients with immunodysregulation, polyendocrinopathy, enteropathy, X-linked syndrome. Bone Marrow Transplant 2014, 49:310-312.
-
(2014)
Bone Marrow Transplant
, vol.49
, pp. 310-312
-
-
Nademi, Z.1
Slatter, M.2
Gambineri, E.3
-
88
-
-
70949098060
-
Combined immunodeficiency associated with DOCK8 mutations
-
Zhang Q., Davis J.C., Lamborn I.T., et al. Combined immunodeficiency associated with DOCK8 mutations. NEngl J Med 2009, 361:2046-2055.
-
(2009)
NEngl J Med
, vol.361
, pp. 2046-2055
-
-
Zhang, Q.1
Davis, J.C.2
Lamborn, I.T.3
-
89
-
-
78649878923
-
Successful engraftment of donor marrow after allogeneic hematopoietic cell transplantation in autosomal-recessive hyper-IgE syndrome caused by dedicator of cytokinesis 8 deficiency
-
McDonald D.R., Massaad M.J., Johnston A., et al. Successful engraftment of donor marrow after allogeneic hematopoietic cell transplantation in autosomal-recessive hyper-IgE syndrome caused by dedicator of cytokinesis 8 deficiency. JAllergy Clin Immunol 2010, 126:1304-1305.e3.
-
(2010)
JAllergy Clin Immunol
, vol.126
, pp. 1304-1305.e3
-
-
McDonald, D.R.1
Massaad, M.J.2
Johnston, A.3
-
90
-
-
78349303895
-
Successful long-term correction of autosomal recessive hyper-IgE syndrome due to DOCK8 deficiency by hematopoietic stem cell transplantation
-
Bittner T.C., Pannicke U., Renner E.D., et al. Successful long-term correction of autosomal recessive hyper-IgE syndrome due to DOCK8 deficiency by hematopoietic stem cell transplantation. Klin Padiatr 2010, 222:351-355.
-
(2010)
Klin Padiatr
, vol.222
, pp. 351-355
-
-
Bittner, T.C.1
Pannicke, U.2
Renner, E.D.3
-
91
-
-
84897563653
-
Haploidentical stem cell transplantation in DOCK8 deficiency - successful control of pre-existing severe viremia with a TCRass/CD19-depleted graft and antiviral treatment
-
Ghosh S., Schuster F.R., Adams O., et al. Haploidentical stem cell transplantation in DOCK8 deficiency - successful control of pre-existing severe viremia with a TCRass/CD19-depleted graft and antiviral treatment. Clin Immunol 2014, 152:111-114.
-
(2014)
Clin Immunol
, vol.152
, pp. 111-114
-
-
Ghosh, S.1
Schuster, F.R.2
Adams, O.3
-
92
-
-
84875704882
-
In DOCK8 deficiency donor cell engraftment post-genoidentical hematopoietic stem cell transplantation is possible without conditioning
-
Al-Mousa H., Hawwari A., Alsum Z. In DOCK8 deficiency donor cell engraftment post-genoidentical hematopoietic stem cell transplantation is possible without conditioning. JAllergy Clin Immunol 2013, 131:1244-1245.
-
(2013)
JAllergy Clin Immunol
, vol.131
, pp. 1244-1245
-
-
Al-Mousa, H.1
Hawwari, A.2
Alsum, Z.3
-
93
-
-
84866945470
-
Clinical and immunological correction of DOCK8 deficiency by allogeneic hematopoietic stem cell transplantation following a reduced toxicity conditioning regimen
-
Boztug H., Karitnig-Weiss C., Ausserer B., et al. Clinical and immunological correction of DOCK8 deficiency by allogeneic hematopoietic stem cell transplantation following a reduced toxicity conditioning regimen. Pediatr Hematol Oncol 2012, 29:585-594.
-
(2012)
Pediatr Hematol Oncol
, vol.29
, pp. 585-594
-
-
Boztug, H.1
Karitnig-Weiss, C.2
Ausserer, B.3
-
94
-
-
79961014871
-
Successful allogeneic hematopoietic stem cell transplantation for DOCK8 deficiency
-
Barlogis V., Galambrun C., Chambost H., et al. Successful allogeneic hematopoietic stem cell transplantation for DOCK8 deficiency. JAllergy Clin Immunol 2011, 128:420-422.e2.
-
(2011)
JAllergy Clin Immunol
, vol.128
, pp. 420-422.e2
-
-
Barlogis, V.1
Galambrun, C.2
Chambost, H.3
-
95
-
-
84861194430
-
Successful bone marrow transplantation for DOCK8 deficient hyper IgE syndrome
-
Metin A., Tavil B., Azik F., et al. Successful bone marrow transplantation for DOCK8 deficient hyper IgE syndrome. Pediatr Transplant 2012, 16:398-399.
-
(2012)
Pediatr Transplant
, vol.16
, pp. 398-399
-
-
Metin, A.1
Tavil, B.2
Azik, F.3
|