-
1
-
-
14144255733
-
Lysosomal storage disorders
-
Vellodi A. Lysosomal storage disorders. Br J Haematol. 2005;128:413-431.
-
(2005)
Br J Haematol
, vol.128
, pp. 413-431
-
-
Vellodi, A.1
-
2
-
-
0027466761
-
Longterm outcome of Hurler syndrome following bone marrow transplantation
-
WhitleyCB. Belani KG, Chang PN.et al. Longterm outcome of Hurler syndrome following bone marrow transplantation. Am J Med Genetics. 1993;46:209-218.
-
(1993)
Am J Med Genetics
, vol.46
, pp. 209-218
-
-
WhitleyCB1
Belani, K.G.2
Chang, P.N.3
-
4
-
-
0034718378
-
Long-term effect of bone-marrow transplantation for childhood-onset cerebral X-linked adrenoleukodystrophy
-
Shapiro E, Krivit W, Lockman L et al. Long-term effect of bone-marrow transplantation for childhood-onset cerebral X-linked adrenoleukodystrophy. Lancet. 2000:356:713-718.
-
(2000)
Lancet
, vol.356
, pp. 713-718
-
-
Shapiro, E.1
Krivit, W.2
Lockman, L.3
-
5
-
-
9344245169
-
Outcome of unrelated donor bone marrow transplantation in 40 children with Hurler syndrome
-
Peters C. Balthazor M, Shapiro EG. et al. Outcome of unrelated donor bone marrow transplantation in 40 children with Hurler syndrome. Blood. 1996:87:4894-4902.
-
(1996)
Blood
, vol.87
, pp. 4894-4902
-
-
Peters, C.1
Balthazor, M.2
Shapiro, E.G.3
-
6
-
-
0032055564
-
Hurler syndrome. II: Outcome of HLA-genotypically identical sibling and HLA-haploidentical related donor bone marrow transplantation in fifty-four children. The Storage Disease Collaborative Study Group
-
Peters C, Shapiro EG, Anderson J, et al. Hurler syndrome. II: outcome of HLA-genotypically identical sibling and HLA-haploidentical related donor bone marrow transplantation in fifty-four children. The Storage Disease Collaborative Study Group. Blood. 1998:91:2601-2608.
-
(1998)
Blood
, vol.91
, pp. 2601-2608
-
-
Peters, C.1
Shapiro, E.G.2
Anderson, J.3
-
7
-
-
3242762263
-
Cerebral X-linked adrenoleukodystrophy: The international hematopoietic cell transplantation experience from 1982 to 1999
-
Peters C. Charnas LR, Tan Y. et al. Cerebral X-linked adrenoleukodystrophy: the international hematopoietic cell transplantation experience from 1982 to 1999. Blood. 2004:104:881-888.
-
(2004)
Blood
, vol.104
, pp. 881-888
-
-
Peters, C.1
Charnas, L.R.2
Tan, Y.3
-
8
-
-
0035029829
-
Four year follow-up of a case of fucosidosis treated with unrelated donor bone marrow transplantation
-
Miano M, Lanino E, Gatti R, et al. Four year follow-up of a case of fucosidosis treated with unrelated donor bone marrow transplantation. Bone Marrow Transplant. 2001 27:747-751.
-
(2001)
Bone Marrow Transplant
, vol.27
, pp. 747-751
-
-
Miano, M.1
Lanino, E.2
Gatti, R.3
-
9
-
-
0030036466
-
Clinical outcome in four children with metachromatic leukodystrophy treated by bone marrow transplantation
-
Malm G, Ringden O, Winiarski J, et al. Clinical outcome in four children with metachromatic leukodystrophy treated by bone marrow transplantation. Bone Marrow Transplant. 1996;17:1003-1008.
-
(1996)
Bone Marrow Transplant
, vol.17
, pp. 1003-1008
-
-
Malm, G.1
Ringden, O.2
Winiarski, J.3
-
10
-
-
0021683336
-
Bonemarrow transplantation in the Maroteaux-Lamy syndrome (mucopolysaccharidosis type VI): Biochemical and clinical status 24 months after transplantation
-
Krivit W, Pierpont ME, AyazK, et al. Bonemarrow transplantation in the Maroteaux-Lamy syndrome (mucopolysaccharidosis type VI): biochemical and clinical status 24 months after transplantation. N Engl J Med. 1984;311:1606-1611.
-
(1984)
N Engl J Med
, vol.311
, pp. 1606-1611
-
-
Krivit, W.1
Pierpont, M.E.2
AyazK3
-
11
-
-
0025022733
-
Treatment of late infantile metachromatic leukodystrophy by bone marrow transplantation
-
Krivit W, Shapiro E, Kennedy W, et al. Treatment of late infantile metachromatic leukodystrophy by bone marrow transplantation. N Engl J Med. 1990:322:28-32.
-
(1990)
N Engl J Med
, vol.322
, pp. 28-32
-
-
Krivit, W.1
Shapiro, E.2
Kennedy, W.3
-
12
-
-
0032537062
-
Hematopoietic stem-cell transplantation in globoid-cell leukodystrophy
-
Krivit W, Shapiro EG, Peters C, et al. Hematopoietic stem-cell transplantation in globoid-cell leukodystrophy. N Engl J Med. 1998;338:1119-1126.
-
(1998)
N Engl J Med
, vol.338
, pp. 1119-1126
-
-
Krivit, W.1
Shapiro, E.G.2
Peters, C.3
-
13
-
-
0019466750
-
Reversal of clinical features of Hurlers disease and biochemical improvement after treatment by bone-marrow transplantation
-
Hobbs JR, Hugh-Jones K, Barrett AJ, et al. Reversal of clinical features of Hurlers disease and biochemical improvement after treatment by bone-marrow transplantation. Lancet. 1981;2: 709-712.
-
(1981)
Lancet
, vol.2
, pp. 709-712
-
-
Hobbs, J.R.1
Hugh-Jones, K.2
Barrett, A.J.3
-
14
-
-
0029013748
-
Juvenile metachromatic leukodystrophy: Neurological outcome two years after bone marrow transplantation
-
Guffon N, Souillet G, Maire I, et al. Juvenile metachromatic leukodystrophy: neurological outcome two years after bone marrow transplantation. J Inherited Metabol Dis. 1995;18:159-161.
-
(1995)
J Inherited Metabol Dis
, vol.18
, pp. 159-161
-
-
Guffon, N.1
Souillet, G.2
Maire, I.3
-
15
-
-
0025329012
-
Reversal of early neurologic and neuroradiologic manifestations of X-linked adrenoleukodystrophy by bone marrow transplantation
-
Aubourg P, Blanche S, Jambaque I, et al. Reversal of early neurologic and neuroradiologic manifestations of X-linked adrenoleukodystrophy by bone marrow transplantation. N Engl J Med. 1990:322:1860-1866.
-
(1990)
N Engl J Med
, vol.322
, pp. 1860-1866
-
-
Aubourg, P.1
Blanche, S.2
Jambaque, I.3
-
16
-
-
38849131982
-
Emerging trends in transplantation of inherited metabolic diseases
-
Prasad VK, Kurtzberg J. Emerging trends in transplantation of inherited metabolic diseases. Bone Marrow Transplant. 2008;41:99-108.
-
(2008)
Bone Marrow Transplant
, vol.41
, pp. 99-108
-
-
Prasad, V.K.1
Kurtzberg, J.2
-
17
-
-
2342524107
-
Bone marrow transdifferentiation in brain after transplantation: A retrospective study
-
Cogle CR, Yachnis AT, Laywell ED, et al. Bone marrow transdifferentiation in brain after transplantation: a retrospective study. Lancet. 2004; 363:1432-1437.
-
(2004)
Lancet
, vol.363
, pp. 1432-1437
-
-
Cogle, C.R.1
Yachnis, A.T.2
Laywell, E.D.3
-
18
-
-
0023949553
-
Perivascular microglial cells of the CNS are bone marrow-derived and present antigen in vivo
-
Hickey WF, Kimura H. Perivascular microglial cells of the CNS are bone marrow-derived and present antigen in vivo. Science. 1988:239:290-292.
-
(1988)
Science
, vol.239
, pp. 290-292
-
-
Hickey, W.F.1
Kimura, H.2
-
19
-
-
0030891163
-
Hematopoietic cells differentiate into both microglia and macroglia in the brains of adult mice
-
Eglitis MA, Mezey E. Hematopoietic cells differentiate into both microglia and macroglia in the brains of adult mice. Proc Natl Acad Sci U S A. 1997;94:4080-4085.
-
(1997)
Proc Natl Acad Sci U S A
, vol.94
, pp. 4080-4085
-
-
Eglitis, M.A.1
Mezey, E.2
-
20
-
-
0035874535
-
Survival after transplantation of unrelated donor umbilical cord blood is comparable to that of human leukocyte antigen-matched unrelated donor bone marrow: Results of a matched-pair analysis
-
Barker JN, Davies SM, Defor T et al. Survival after transplantation of unrelated donor umbilical cord blood is comparable to that of human leukocyte antigen-matched unrelated donor bone marrow: results of a matched-pair analysis. Blood. 2001;97:2957-2961.
-
(2001)
Blood
, vol.97
, pp. 2957-2961
-
-
Barker, J.N.1
Davies, S.M.2
Defor, T.3
-
21
-
-
0025881307
-
Umbilical cord blood hematopoietic stem and repopulating cells in human clinical transplantation
-
Broxmeyer HE, Kurtzberg J, Gluckman E, et al. Umbilical cord blood hematopoietic stem and repopulating cells in human clinical transplantation. Blood Cells. 1991;17:313-329.
-
(1991)
Blood Cells
, vol.17
, pp. 313-329
-
-
Broxmeyer, H.E.1
Kurtzberg, J.2
Gluckman, E.3
-
22
-
-
20844453744
-
Transplantation of umbilical-cord blood in babies with infantile Krabbe's disease
-
Escolar ML, Poe MD, Provenzale JM, et al. Transplantation of umbilical-cord blood in babies with infantile Krabbe's disease. N Engl J Med. 2005;352:2069-2081.
-
(2005)
N Engl J Med
, vol.352
, pp. 2069-2081
-
-
Escolar, M.L.1
Poe, M.D.2
Provenzale, J.M.3
-
23
-
-
0024396816
-
Hematopoietic reconstitution in a patient with Fanconi's anemia by means of umbilical-cord blood from an HLA-identical sibling
-
Gluckman E, Broxmeyer HA. Auerbach AD, et al. Hematopoietic reconstitution in a patient with Fanconi's anemia by means of umbilical-cord blood from an HLA-identical sibling. N Engl J Med. 1989:321:1174-1178.
-
(1989)
N Engl J Med
, vol.321
, pp. 1174-1178
-
-
Gluckman, E.1
Broxmeyer, H.A.2
Auerbach, A.D.3
-
24
-
-
0029997538
-
Bone marrow transplantation in children with hereditary disorders [review]
-
Gluckman E. Bone marrow transplantation in children with hereditary disorders [review]. Curr Opin Pediatr. 1996;8:42-44.
-
(1996)
Curr Opin Pediatr
, vol.8
, pp. 42-44
-
-
Gluckman, E.1
-
25
-
-
8944228913
-
Placental blood as a source of hematopoietic stem cells for transplantation into unrelated recipients
-
Kurtzberg J, Laughlin M, Graham ML, et al. Placental blood as a source of hematopoietic stem cells for transplantation into unrelated recipients. N Engl J Med. 1996;335:157-166.
-
(1996)
N Engl J Med
, vol.335
, pp. 157-166
-
-
Kurtzberg, J.1
Laughlin, M.2
Graham, M.L.3
-
26
-
-
31344470473
-
Results of the cord blood transplantation study (COBLT): Outcomes of unrelated donor umbilical cord blood transplantation in pediatric patients with lysosomal and peroxisomal storage diseases
-
Martin PL, Carter SL, Kernan NA, et al. Results of the cord blood transplantation study (COBLT): outcomes of unrelated donor umbilical cord blood transplantation in pediatric patients with lysosomal and peroxisomal storage diseases. Biol Blood Marrow Transplant. 2006; 12:184-194.
-
(2006)
Biol Blood Marrow Transplant
, vol.12
, pp. 184-194
-
-
Martin, P.L.1
Carter, S.L.2
Kernan, N.A.3
-
27
-
-
0035874490
-
Comparison of outcomes of unrelated bone marrow and umbilical cord blood transplants in children with acute leukemia
-
Rocha V, Cornish J, Sievers EL. et al. Comparison of outcomes of unrelated bone marrow and umbilical cord blood transplants in children with acute leukemia. Blood. 2001;97:2962-2971.
-
(2001)
Blood
, vol.97
, pp. 2962-2971
-
-
Rocha, V.1
Cornish, J.2
Sievers, E.L.3
-
28
-
-
0032569779
-
Outcomes among 562 recipients of placental-blood transplants from unrelated donors
-
Rubinstein P, Carrier C, ScaradavouA, et al. Outcomes among 562 recipients of placental-blood transplants from unrelated donors. N Engl J Med. 1998:339:1565-1577.
-
(1998)
N Engl J Med
, vol.339
, pp. 1565-1577
-
-
Rubinstein, P.1
Carrier, C.2
ScaradavouA3
-
29
-
-
0029742950
-
Successful transplantation of HLA-matched and HLA-mismatched umbilical cord blood from unrelated donors: Analysis of engraftment and acute graft-versus-host disease
-
Wagner JE, Rosenthal J, Sweetman R, et al. Successful transplantation of HLA-matched and HLA-mismatched umbilical cord blood from unrelated donors: analysis of engraftment and acute graft-versus-host disease. Blood. 1996; 88:795-802.
-
(1996)
Blood
, vol.88
, pp. 795-802
-
-
Wagner, J.E.1
Rosenthal, J.2
Sweetman, R.3
-
30
-
-
34248668484
-
Outcomes of unrelated umbilical cord blood transplantation for x-linked adrenoleukodystrophy
-
Beam D, Poe MD, Provenzale JM, et al. Outcomes of unrelated umbilical cord blood transplantation for x-linked adrenoleukodystrophy. Biol Blood Marrow Transplant. 2007:13:665-674.
-
(2007)
Biol Blood Marrow Transplant
, vol.13
, pp. 665-674
-
-
Beam, D.1
Poe, M.D.2
Provenzale, J.M.3
-
31
-
-
2342535103
-
Cord-blood transplants from unrelated donors in patients with Hurlers syndrome
-
Staba SL, Escolar ML, Poe M et al. Cord-blood transplants from unrelated donors in patients with Hurlers syndrome. N Engl J Med. 2004;350: 1960-1969.
-
(2004)
N Engl J Med
, vol.350
, pp. 1960-1969
-
-
Staba, S.L.1
Escolar, M.L.2
Poe, M.3
-
33
-
-
0034108263
-
Measurements from normal umbilical cord blood of four lysosomal enzymatic activities: Alpha-L-iduronidase (Hurler), galactocerebrosidase (globoid cell leukodystrophy), arylsulfatase A (metachromatic leukodystrophy), arylsulfatase B (Maroteaux-Lamy)
-
DeGasperi R, Raghavan SS, Sosa MG, et al. Measurements from normal umbilical cord blood of four lysosomal enzymatic activities: alpha-L-iduronidase (Hurler), galactocerebrosidase (globoid cell leukodystrophy), arylsulfatase A (metachromatic leukodystrophy), arylsulfatase B (Maroteaux-Lamy). Bone Marrow Transplant. 2000;25:541-544.
-
(2000)
Bone Marrow Transplant
, vol.25
, pp. 541-544
-
-
DeGasperi, R.1
Raghavan, S.S.2
Sosa, M.G.3
-
34
-
-
0028785797
-
Processing and cryopreservation of placental/umbilical cord blood for unrelated bone marrow reconstitution
-
Rubinstein P, Dobrila L, Rosenfield RE, et al. Processing and cryopreservation of placental/umbilical cord blood for unrelated bone marrow reconstitution. Proc Natl Acad Sci U S A. 1995;92: 10119-10122.
-
(1995)
Proc Natl Acad Sci U S A
, vol.92
, pp. 10119-10122
-
-
Rubinstein, P.1
Dobrila, L.2
Rosenfield, R.E.3
-
36
-
-
28744444180
-
National Institutes of Health consensus development project on criteria for clinical trials in chronic graft-versus-host disease. I: Diagnosis and staging working group report
-
Filipovich AH, Weisdorf D, Pavletic S, et al. National Institutes of Health consensus development project on criteria for clinical trials in chronic graft-versus-host disease. I: diagnosis and staging working group report. Biol Blood Marrow Transplant. 2005; 11:945-956.
-
(2005)
Biol Blood Marrow Transplant
, vol.11
, pp. 945-956
-
-
Filipovich, A.H.1
Weisdorf, D.2
Pavletic, S.3
-
37
-
-
0033616908
-
Estimation of failure probabilities in the presence of competing risks: New representations of old estimators
-
Gooley TA. Leisenring W. Crowley J. Storer BE. Estimation of failure probabilities in the presence of competing risks: new representations of old estimators. Stat Med. 1999:18:695-706.
-
(1999)
Stat Med
, vol.18
, pp. 695-706
-
-
Gooley, T.A.1
Leisenring, W.2
Crowley, J.3
Storer, B.E.4
-
38
-
-
0000120995
-
A class of K-sample tests for comparing the cumulative incidence of a competing risk
-
Gray RJ. A class of K-sample tests for comparing the cumulative incidence of a competing risk. Ann Stat. 1988:16:1141-1154.
-
(1988)
Ann Stat
, vol.16
, pp. 1141-1154
-
-
Gray, R.J.1
-
39
-
-
33845382806
-
Nonparametric estimation from incomplete observations
-
Kaplan EL. Meier P. Nonparametric estimation from incomplete observations. J Am Stat Assoc. 1958:53:457-481.
-
(1958)
J Am Stat Assoc
, vol.53
, pp. 457-481
-
-
Kaplan, E.L.1
Meier, P.2
-
40
-
-
84959801619
-
Statistical aspects of the analysis of data from retrospective studies of disease
-
Mantel N, Haenszel W. Statistical aspects of the analysis of data from retrospective studies of disease. J Natl Cancer Inst. 1959:22:719-748.
-
(1959)
J Natl Cancer Inst
, vol.22
, pp. 719-748
-
-
Mantel, N.1
Haenszel, W.2
-
41
-
-
0000336139
-
Regression models and life-tables
-
Cox DR. Regression models and life-tables. J Royal Stat Soc Series B. 1972:34:187-220.
-
(1972)
J Royal Stat Soc Series B
, vol.34
, pp. 187-220
-
-
Cox, D.R.1
-
42
-
-
22344435686
-
Busulfan/melphalan/antithymocyte globulin followed by unrelated donor cord blood transplantation for treatment of infant leukemia and leukemia in young children: The Cord Blood Transplantation study (COBLT) experience
-
Wall DA, Carter SL. Kernan NA, et al. Busulfan/melphalan/antithymocyte globulin followed by unrelated donor cord blood transplantation for treatment of infant leukemia and leukemia in young children: the Cord Blood Transplantation study (COBLT) experience. Biol Blood Marrow Transplant. 2005; 11:637-646.
-
(2005)
Biol Blood Marrow Transplant
, vol.11
, pp. 637-646
-
-
Wall, D.A.1
Carter, S.L.2
Kernan, N.A.3
-
43
-
-
61849128543
-
Post transplant autoimmune hemolytic anemia and other cytopenias are increased in young babies undergoing unrelated donor umbilical cord blood transplantation
-
Page KM, Wood S, Prasad VK, Szabolcs P, Kurtzberg J. Post transplant autoimmune hemolytic anemia and other cytopenias are increased in young babies undergoing unrelated donor umbilical cord blood transplantation. Biol Blood Marrow Transplant. 2007; 13:64.
-
(2007)
Biol Blood Marrow Transplant
, vol.13
, pp. 64
-
-
Page, K.M.1
Wood, S.2
Prasad, V.K.3
Szabolcs, P.4
Kurtzberg, J.5
-
44
-
-
33745099961
-
Trends in haematopoietic cell transplantation for inborn errors of metabolism [review]
-
Boelens JJ. Trends in haematopoietic cell transplantation for inborn errors of metabolism [review]. J Inherited Metabol Dis. 2006:29:413-420.
-
(2006)
J Inherited Metabol Dis
, vol.29
, pp. 413-420
-
-
Boelens, J.J.1
-
45
-
-
34547147899
-
Outcomes of hematopoietic stem cell transplantation for Hurlers syndrome in Europe: A risk factor analysis for graft failure
-
Boelens JJ. Wynn RF. O'Meara A. et al. Outcomes of hematopoietic stem cell transplantation for Hurlers syndrome in Europe: a risk factor analysis for graft failure. Bone Marrow Transplant. 2007:40:225-233.
-
(2007)
Bone Marrow Transplant
, vol.40
, pp. 225-233
-
-
Boelens, J.J.1
Wynn, R.F.2
O'Meara, A.3
-
46
-
-
10744223978
-
Outcome of 27 patients with Hurlers syndrome transplanted from either related or unrelated haematopoietic stem cell sources
-
SouilletG, Guffon N, Maire I, et al. Outcome of 27 patients with Hurlers syndrome transplanted from either related or unrelated haematopoietic stem cell sources. Bone Marrow Transplant. 2003:31:1105-1117.
-
(2003)
Bone Marrow Transplant
, vol.31
, pp. 1105-1117
-
-
Souillet, G.1
Guffon, N.2
Maire, I.3
|