Hematopoietic Stem Cell Transplantation for Children With Inborn Errors of Metabolism: Single Center Experience Over Two Decades
Objective We present outcome data on hematopoietic stem cell transplantation (HSCT) in children with inborn errors of metabolism (IEM). Methods We retrospectively analyzed data on children up to 18 years of age, diagnosed with IEM, who underwent HSCT between January, 2002 and December, 2020. Results...
Ausführliche Beschreibung
Gespeichert in:
| Autor*in: |
Swaminathan, Venkateswaran Vellaichamy [verfasserIn] |
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| Format: |
E-Artikel |
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| Sprache: |
Englisch |
| Erschienen: |
2022 |
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| Schlagwörter: |
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| Anmerkung: |
© Indian Academy of Pediatrics 2022 |
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| Übergeordnetes Werk: |
Enthalten in: Indian Pediatrics - Springer-Verlag, 2010, 59(2022), 9 vom: 27. Juni, Seite 699-702 |
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| Übergeordnetes Werk: |
volume:59 ; year:2022 ; number:9 ; day:27 ; month:06 ; pages:699-702 |
| Links: |
|---|
| DOI / URN: |
10.1007/s13312-022-2597-z |
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| Katalog-ID: |
SPR048238287 |
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| 100 | 1 | |a Swaminathan, Venkateswaran Vellaichamy |e verfasserin |4 aut | |
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| 520 | |a Objective We present outcome data on hematopoietic stem cell transplantation (HSCT) in children with inborn errors of metabolism (IEM). Methods We retrospectively analyzed data on children up to 18 years of age, diagnosed with IEM, who underwent HSCT between January, 2002 and December, 2020. Results 24 children, (mucopolysaccharidosis — 13, Gaucher disease — 4, X-linked adrenoleukodystrophy — 4, metachromatic leukodystrophy — 2, Krabbe disease — 1) were included. Donors were matched family donors in 24%, matched unrelated donors in 34%, and haploidentical fathers in 42% of the transplants, with engraftment in 91% of children. Overall survival was 72% (55–100%) with a median follow-up of 76.5 (10–120) months, and progression-free survival of 68% (MPS-76%, X-ALD — 60%, Gaucher disease — 50%, and 100% in MLD and Krabbe disease). Conclusion HSCT is an available curative option, and early age at HSCT prevents end-organ damage. | ||
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| 700 | 1 | |a Chandar, Rumesh |4 aut | |
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| 700 | 1 | |a Ramakrishnan, Balasubramaniam |4 aut | |
| 700 | 1 | |a Uppuluri, Ramya |4 aut | |
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10.1007/s13312-022-2597-z doi (DE-627)SPR048238287 (SPR)s13312-022-2597-z-e DE-627 ger DE-627 rakwb eng Swaminathan, Venkateswaran Vellaichamy verfasserin aut Hematopoietic Stem Cell Transplantation for Children With Inborn Errors of Metabolism: Single Center Experience Over Two Decades 2022 Text txt rdacontent Computermedien c rdamedia Online-Ressource cr rdacarrier © Indian Academy of Pediatrics 2022 Objective We present outcome data on hematopoietic stem cell transplantation (HSCT) in children with inborn errors of metabolism (IEM). Methods We retrospectively analyzed data on children up to 18 years of age, diagnosed with IEM, who underwent HSCT between January, 2002 and December, 2020. Results 24 children, (mucopolysaccharidosis — 13, Gaucher disease — 4, X-linked adrenoleukodystrophy — 4, metachromatic leukodystrophy — 2, Krabbe disease — 1) were included. Donors were matched family donors in 24%, matched unrelated donors in 34%, and haploidentical fathers in 42% of the transplants, with engraftment in 91% of children. Overall survival was 72% (55–100%) with a median follow-up of 76.5 (10–120) months, and progression-free survival of 68% (MPS-76%, X-ALD — 60%, Gaucher disease — 50%, and 100% in MLD and Krabbe disease). Conclusion HSCT is an available curative option, and early age at HSCT prevents end-organ damage. Alternate donor HSCT (dpeaa)DE-He213 Gaucher disease (dpeaa)DE-He213 Leukodystrophy (dpeaa)DE-He213 Mucopolysaccharidosis (dpeaa)DE-He213 Meena, Satishkumar aut Varla, Harika aut Chandar, Rumesh aut Jayakumar, Indira aut Ramakrishnan, Balasubramaniam aut Uppuluri, Ramya aut Raj, Revathi aut Enthalten in Indian Pediatrics Springer-Verlag, 2010 59(2022), 9 vom: 27. Juni, Seite 699-702 (DE-627)SPR031274943 nnns volume:59 year:2022 number:9 day:27 month:06 pages:699-702 https://dx.doi.org/10.1007/s13312-022-2597-z lizenzpflichtig Volltext GBV_USEFLAG_A SYSFLAG_A GBV_SPRINGER SSG-OLC-PHA GBV_ILN_11 GBV_ILN_20 GBV_ILN_22 GBV_ILN_70 GBV_ILN_72 GBV_ILN_110 GBV_ILN_131 GBV_ILN_160 GBV_ILN_376 GBV_ILN_607 AR 59 2022 9 27 06 699-702 |
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10.1007/s13312-022-2597-z doi (DE-627)SPR048238287 (SPR)s13312-022-2597-z-e DE-627 ger DE-627 rakwb eng Swaminathan, Venkateswaran Vellaichamy verfasserin aut Hematopoietic Stem Cell Transplantation for Children With Inborn Errors of Metabolism: Single Center Experience Over Two Decades 2022 Text txt rdacontent Computermedien c rdamedia Online-Ressource cr rdacarrier © Indian Academy of Pediatrics 2022 Objective We present outcome data on hematopoietic stem cell transplantation (HSCT) in children with inborn errors of metabolism (IEM). Methods We retrospectively analyzed data on children up to 18 years of age, diagnosed with IEM, who underwent HSCT between January, 2002 and December, 2020. Results 24 children, (mucopolysaccharidosis — 13, Gaucher disease — 4, X-linked adrenoleukodystrophy — 4, metachromatic leukodystrophy — 2, Krabbe disease — 1) were included. Donors were matched family donors in 24%, matched unrelated donors in 34%, and haploidentical fathers in 42% of the transplants, with engraftment in 91% of children. Overall survival was 72% (55–100%) with a median follow-up of 76.5 (10–120) months, and progression-free survival of 68% (MPS-76%, X-ALD — 60%, Gaucher disease — 50%, and 100% in MLD and Krabbe disease). Conclusion HSCT is an available curative option, and early age at HSCT prevents end-organ damage. Alternate donor HSCT (dpeaa)DE-He213 Gaucher disease (dpeaa)DE-He213 Leukodystrophy (dpeaa)DE-He213 Mucopolysaccharidosis (dpeaa)DE-He213 Meena, Satishkumar aut Varla, Harika aut Chandar, Rumesh aut Jayakumar, Indira aut Ramakrishnan, Balasubramaniam aut Uppuluri, Ramya aut Raj, Revathi aut Enthalten in Indian Pediatrics Springer-Verlag, 2010 59(2022), 9 vom: 27. Juni, Seite 699-702 (DE-627)SPR031274943 nnns volume:59 year:2022 number:9 day:27 month:06 pages:699-702 https://dx.doi.org/10.1007/s13312-022-2597-z lizenzpflichtig Volltext GBV_USEFLAG_A SYSFLAG_A GBV_SPRINGER SSG-OLC-PHA GBV_ILN_11 GBV_ILN_20 GBV_ILN_22 GBV_ILN_70 GBV_ILN_72 GBV_ILN_110 GBV_ILN_131 GBV_ILN_160 GBV_ILN_376 GBV_ILN_607 AR 59 2022 9 27 06 699-702 |
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10.1007/s13312-022-2597-z doi (DE-627)SPR048238287 (SPR)s13312-022-2597-z-e DE-627 ger DE-627 rakwb eng Swaminathan, Venkateswaran Vellaichamy verfasserin aut Hematopoietic Stem Cell Transplantation for Children With Inborn Errors of Metabolism: Single Center Experience Over Two Decades 2022 Text txt rdacontent Computermedien c rdamedia Online-Ressource cr rdacarrier © Indian Academy of Pediatrics 2022 Objective We present outcome data on hematopoietic stem cell transplantation (HSCT) in children with inborn errors of metabolism (IEM). Methods We retrospectively analyzed data on children up to 18 years of age, diagnosed with IEM, who underwent HSCT between January, 2002 and December, 2020. Results 24 children, (mucopolysaccharidosis — 13, Gaucher disease — 4, X-linked adrenoleukodystrophy — 4, metachromatic leukodystrophy — 2, Krabbe disease — 1) were included. Donors were matched family donors in 24%, matched unrelated donors in 34%, and haploidentical fathers in 42% of the transplants, with engraftment in 91% of children. Overall survival was 72% (55–100%) with a median follow-up of 76.5 (10–120) months, and progression-free survival of 68% (MPS-76%, X-ALD — 60%, Gaucher disease — 50%, and 100% in MLD and Krabbe disease). Conclusion HSCT is an available curative option, and early age at HSCT prevents end-organ damage. Alternate donor HSCT (dpeaa)DE-He213 Gaucher disease (dpeaa)DE-He213 Leukodystrophy (dpeaa)DE-He213 Mucopolysaccharidosis (dpeaa)DE-He213 Meena, Satishkumar aut Varla, Harika aut Chandar, Rumesh aut Jayakumar, Indira aut Ramakrishnan, Balasubramaniam aut Uppuluri, Ramya aut Raj, Revathi aut Enthalten in Indian Pediatrics Springer-Verlag, 2010 59(2022), 9 vom: 27. Juni, Seite 699-702 (DE-627)SPR031274943 nnns volume:59 year:2022 number:9 day:27 month:06 pages:699-702 https://dx.doi.org/10.1007/s13312-022-2597-z lizenzpflichtig Volltext GBV_USEFLAG_A SYSFLAG_A GBV_SPRINGER SSG-OLC-PHA GBV_ILN_11 GBV_ILN_20 GBV_ILN_22 GBV_ILN_70 GBV_ILN_72 GBV_ILN_110 GBV_ILN_131 GBV_ILN_160 GBV_ILN_376 GBV_ILN_607 AR 59 2022 9 27 06 699-702 |
| allfieldsGer |
10.1007/s13312-022-2597-z doi (DE-627)SPR048238287 (SPR)s13312-022-2597-z-e DE-627 ger DE-627 rakwb eng Swaminathan, Venkateswaran Vellaichamy verfasserin aut Hematopoietic Stem Cell Transplantation for Children With Inborn Errors of Metabolism: Single Center Experience Over Two Decades 2022 Text txt rdacontent Computermedien c rdamedia Online-Ressource cr rdacarrier © Indian Academy of Pediatrics 2022 Objective We present outcome data on hematopoietic stem cell transplantation (HSCT) in children with inborn errors of metabolism (IEM). Methods We retrospectively analyzed data on children up to 18 years of age, diagnosed with IEM, who underwent HSCT between January, 2002 and December, 2020. Results 24 children, (mucopolysaccharidosis — 13, Gaucher disease — 4, X-linked adrenoleukodystrophy — 4, metachromatic leukodystrophy — 2, Krabbe disease — 1) were included. Donors were matched family donors in 24%, matched unrelated donors in 34%, and haploidentical fathers in 42% of the transplants, with engraftment in 91% of children. Overall survival was 72% (55–100%) with a median follow-up of 76.5 (10–120) months, and progression-free survival of 68% (MPS-76%, X-ALD — 60%, Gaucher disease — 50%, and 100% in MLD and Krabbe disease). Conclusion HSCT is an available curative option, and early age at HSCT prevents end-organ damage. Alternate donor HSCT (dpeaa)DE-He213 Gaucher disease (dpeaa)DE-He213 Leukodystrophy (dpeaa)DE-He213 Mucopolysaccharidosis (dpeaa)DE-He213 Meena, Satishkumar aut Varla, Harika aut Chandar, Rumesh aut Jayakumar, Indira aut Ramakrishnan, Balasubramaniam aut Uppuluri, Ramya aut Raj, Revathi aut Enthalten in Indian Pediatrics Springer-Verlag, 2010 59(2022), 9 vom: 27. Juni, Seite 699-702 (DE-627)SPR031274943 nnns volume:59 year:2022 number:9 day:27 month:06 pages:699-702 https://dx.doi.org/10.1007/s13312-022-2597-z lizenzpflichtig Volltext GBV_USEFLAG_A SYSFLAG_A GBV_SPRINGER SSG-OLC-PHA GBV_ILN_11 GBV_ILN_20 GBV_ILN_22 GBV_ILN_70 GBV_ILN_72 GBV_ILN_110 GBV_ILN_131 GBV_ILN_160 GBV_ILN_376 GBV_ILN_607 AR 59 2022 9 27 06 699-702 |
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10.1007/s13312-022-2597-z doi (DE-627)SPR048238287 (SPR)s13312-022-2597-z-e DE-627 ger DE-627 rakwb eng Swaminathan, Venkateswaran Vellaichamy verfasserin aut Hematopoietic Stem Cell Transplantation for Children With Inborn Errors of Metabolism: Single Center Experience Over Two Decades 2022 Text txt rdacontent Computermedien c rdamedia Online-Ressource cr rdacarrier © Indian Academy of Pediatrics 2022 Objective We present outcome data on hematopoietic stem cell transplantation (HSCT) in children with inborn errors of metabolism (IEM). Methods We retrospectively analyzed data on children up to 18 years of age, diagnosed with IEM, who underwent HSCT between January, 2002 and December, 2020. Results 24 children, (mucopolysaccharidosis — 13, Gaucher disease — 4, X-linked adrenoleukodystrophy — 4, metachromatic leukodystrophy — 2, Krabbe disease — 1) were included. Donors were matched family donors in 24%, matched unrelated donors in 34%, and haploidentical fathers in 42% of the transplants, with engraftment in 91% of children. Overall survival was 72% (55–100%) with a median follow-up of 76.5 (10–120) months, and progression-free survival of 68% (MPS-76%, X-ALD — 60%, Gaucher disease — 50%, and 100% in MLD and Krabbe disease). Conclusion HSCT is an available curative option, and early age at HSCT prevents end-organ damage. Alternate donor HSCT (dpeaa)DE-He213 Gaucher disease (dpeaa)DE-He213 Leukodystrophy (dpeaa)DE-He213 Mucopolysaccharidosis (dpeaa)DE-He213 Meena, Satishkumar aut Varla, Harika aut Chandar, Rumesh aut Jayakumar, Indira aut Ramakrishnan, Balasubramaniam aut Uppuluri, Ramya aut Raj, Revathi aut Enthalten in Indian Pediatrics Springer-Verlag, 2010 59(2022), 9 vom: 27. Juni, Seite 699-702 (DE-627)SPR031274943 nnns volume:59 year:2022 number:9 day:27 month:06 pages:699-702 https://dx.doi.org/10.1007/s13312-022-2597-z lizenzpflichtig Volltext GBV_USEFLAG_A SYSFLAG_A GBV_SPRINGER SSG-OLC-PHA GBV_ILN_11 GBV_ILN_20 GBV_ILN_22 GBV_ILN_70 GBV_ILN_72 GBV_ILN_110 GBV_ILN_131 GBV_ILN_160 GBV_ILN_376 GBV_ILN_607 AR 59 2022 9 27 06 699-702 |
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Swaminathan, Venkateswaran Vellaichamy |
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Swaminathan, Venkateswaran Vellaichamy misc Alternate donor HSCT misc Gaucher disease misc Leukodystrophy misc Mucopolysaccharidosis Hematopoietic Stem Cell Transplantation for Children With Inborn Errors of Metabolism: Single Center Experience Over Two Decades |
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Hematopoietic Stem Cell Transplantation for Children With Inborn Errors of Metabolism: Single Center Experience Over Two Decades Alternate donor HSCT (dpeaa)DE-He213 Gaucher disease (dpeaa)DE-He213 Leukodystrophy (dpeaa)DE-He213 Mucopolysaccharidosis (dpeaa)DE-He213 |
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Hematopoietic Stem Cell Transplantation for Children With Inborn Errors of Metabolism: Single Center Experience Over Two Decades |
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Hematopoietic Stem Cell Transplantation for Children With Inborn Errors of Metabolism: Single Center Experience Over Two Decades |
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Swaminathan, Venkateswaran Vellaichamy Meena, Satishkumar Varla, Harika Chandar, Rumesh Jayakumar, Indira Ramakrishnan, Balasubramaniam Uppuluri, Ramya Raj, Revathi |
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hematopoietic stem cell transplantation for children with inborn errors of metabolism: single center experience over two decades |
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Hematopoietic Stem Cell Transplantation for Children With Inborn Errors of Metabolism: Single Center Experience Over Two Decades |
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Objective We present outcome data on hematopoietic stem cell transplantation (HSCT) in children with inborn errors of metabolism (IEM). Methods We retrospectively analyzed data on children up to 18 years of age, diagnosed with IEM, who underwent HSCT between January, 2002 and December, 2020. Results 24 children, (mucopolysaccharidosis — 13, Gaucher disease — 4, X-linked adrenoleukodystrophy — 4, metachromatic leukodystrophy — 2, Krabbe disease — 1) were included. Donors were matched family donors in 24%, matched unrelated donors in 34%, and haploidentical fathers in 42% of the transplants, with engraftment in 91% of children. Overall survival was 72% (55–100%) with a median follow-up of 76.5 (10–120) months, and progression-free survival of 68% (MPS-76%, X-ALD — 60%, Gaucher disease — 50%, and 100% in MLD and Krabbe disease). Conclusion HSCT is an available curative option, and early age at HSCT prevents end-organ damage. © Indian Academy of Pediatrics 2022 |
| abstractGer |
Objective We present outcome data on hematopoietic stem cell transplantation (HSCT) in children with inborn errors of metabolism (IEM). Methods We retrospectively analyzed data on children up to 18 years of age, diagnosed with IEM, who underwent HSCT between January, 2002 and December, 2020. Results 24 children, (mucopolysaccharidosis — 13, Gaucher disease — 4, X-linked adrenoleukodystrophy — 4, metachromatic leukodystrophy — 2, Krabbe disease — 1) were included. Donors were matched family donors in 24%, matched unrelated donors in 34%, and haploidentical fathers in 42% of the transplants, with engraftment in 91% of children. Overall survival was 72% (55–100%) with a median follow-up of 76.5 (10–120) months, and progression-free survival of 68% (MPS-76%, X-ALD — 60%, Gaucher disease — 50%, and 100% in MLD and Krabbe disease). Conclusion HSCT is an available curative option, and early age at HSCT prevents end-organ damage. © Indian Academy of Pediatrics 2022 |
| abstract_unstemmed |
Objective We present outcome data on hematopoietic stem cell transplantation (HSCT) in children with inborn errors of metabolism (IEM). Methods We retrospectively analyzed data on children up to 18 years of age, diagnosed with IEM, who underwent HSCT between January, 2002 and December, 2020. Results 24 children, (mucopolysaccharidosis — 13, Gaucher disease — 4, X-linked adrenoleukodystrophy — 4, metachromatic leukodystrophy — 2, Krabbe disease — 1) were included. Donors were matched family donors in 24%, matched unrelated donors in 34%, and haploidentical fathers in 42% of the transplants, with engraftment in 91% of children. Overall survival was 72% (55–100%) with a median follow-up of 76.5 (10–120) months, and progression-free survival of 68% (MPS-76%, X-ALD — 60%, Gaucher disease — 50%, and 100% in MLD and Krabbe disease). Conclusion HSCT is an available curative option, and early age at HSCT prevents end-organ damage. © Indian Academy of Pediatrics 2022 |
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Hematopoietic Stem Cell Transplantation for Children With Inborn Errors of Metabolism: Single Center Experience Over Two Decades |
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