Successful autologous stem cell transplantation in two patients with juvenile dermatomyositis

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Successful autologous stem cell transplantation in two patients with juvenile dermatomyositis. / Holzer, U; van Royen-Kerkhof, A; van der Torre, P; Kuemmerle-Deschner, J; Well, C; Handgretinger, R; Müller, Ingo; Wulffraat, N.

In: SCAND J RHEUMATOL, Vol. 39, No. 1, 2010, p. 88-92.

Research output: SCORING: Contribution to journalSCORING: Journal articleResearchpeer-review

Harvard

Holzer, U, van Royen-Kerkhof, A, van der Torre, P, Kuemmerle-Deschner, J, Well, C, Handgretinger, R, Müller, I & Wulffraat, N 2010, 'Successful autologous stem cell transplantation in two patients with juvenile dermatomyositis', SCAND J RHEUMATOL, vol. 39, no. 1, pp. 88-92. https://doi.org/10.3109/03009740903096622

APA

Holzer, U., van Royen-Kerkhof, A., van der Torre, P., Kuemmerle-Deschner, J., Well, C., Handgretinger, R., Müller, I., & Wulffraat, N. (2010). Successful autologous stem cell transplantation in two patients with juvenile dermatomyositis. SCAND J RHEUMATOL, 39(1), 88-92. https://doi.org/10.3109/03009740903096622

Vancouver

Holzer U, van Royen-Kerkhof A, van der Torre P, Kuemmerle-Deschner J, Well C, Handgretinger R et al. Successful autologous stem cell transplantation in two patients with juvenile dermatomyositis. SCAND J RHEUMATOL. 2010;39(1):88-92. https://doi.org/10.3109/03009740903096622

Bibtex

@article{37aaf2c6adfa4ad79425885d35d172f3,
title = "Successful autologous stem cell transplantation in two patients with juvenile dermatomyositis",
abstract = "Juvenile dermatomyositis (JDM) is a chronic inflammatory disorder of unknown aetiology that affects muscle and skin. We report on two patients with severe progressive JDM who developed contractures and were wheelchair dependent despite therapy including methotrexate (MTX), steroids, immunoglobulins, cyclosporin A, and rituximab. On account of the refractory disease, autologous stem cell transplantation (ASCT) was performed using a CD3/CD19-depleted graft after immunoablative conditioning with fludarabine, cyclophosphamide, and anti-thymocyte globulin. This induced a dramatic improvement and sustained remission of the disease in both patients. We demonstrate that ASCT is a therapeutic option with low toxicity for patients with severe, refractory JDM.",
keywords = "Child, Dermatomyositis, Female, Follow-Up Studies, Graft Survival, Hematopoietic Stem Cell Transplantation, Humans, Magnetic Resonance Imaging, Pain Measurement, Risk Assessment, Severity of Illness Index, Time Factors, Transplantation Conditioning, Transplantation, Autologous, Treatment Outcome",
author = "U Holzer and {van Royen-Kerkhof}, A and {van der Torre}, P and J Kuemmerle-Deschner and C Well and R Handgretinger and Ingo M{\"u}ller and N Wulffraat",
year = "2010",
doi = "10.3109/03009740903096622",
language = "English",
volume = "39",
pages = "88--92",
journal = "SCAND J RHEUMATOL",
issn = "0300-9742",
publisher = "informa healthcare",
number = "1",

}

RIS

TY - JOUR

T1 - Successful autologous stem cell transplantation in two patients with juvenile dermatomyositis

AU - Holzer, U

AU - van Royen-Kerkhof, A

AU - van der Torre, P

AU - Kuemmerle-Deschner, J

AU - Well, C

AU - Handgretinger, R

AU - Müller, Ingo

AU - Wulffraat, N

PY - 2010

Y1 - 2010

N2 - Juvenile dermatomyositis (JDM) is a chronic inflammatory disorder of unknown aetiology that affects muscle and skin. We report on two patients with severe progressive JDM who developed contractures and were wheelchair dependent despite therapy including methotrexate (MTX), steroids, immunoglobulins, cyclosporin A, and rituximab. On account of the refractory disease, autologous stem cell transplantation (ASCT) was performed using a CD3/CD19-depleted graft after immunoablative conditioning with fludarabine, cyclophosphamide, and anti-thymocyte globulin. This induced a dramatic improvement and sustained remission of the disease in both patients. We demonstrate that ASCT is a therapeutic option with low toxicity for patients with severe, refractory JDM.

AB - Juvenile dermatomyositis (JDM) is a chronic inflammatory disorder of unknown aetiology that affects muscle and skin. We report on two patients with severe progressive JDM who developed contractures and were wheelchair dependent despite therapy including methotrexate (MTX), steroids, immunoglobulins, cyclosporin A, and rituximab. On account of the refractory disease, autologous stem cell transplantation (ASCT) was performed using a CD3/CD19-depleted graft after immunoablative conditioning with fludarabine, cyclophosphamide, and anti-thymocyte globulin. This induced a dramatic improvement and sustained remission of the disease in both patients. We demonstrate that ASCT is a therapeutic option with low toxicity for patients with severe, refractory JDM.

KW - Child

KW - Dermatomyositis

KW - Female

KW - Follow-Up Studies

KW - Graft Survival

KW - Hematopoietic Stem Cell Transplantation

KW - Humans

KW - Magnetic Resonance Imaging

KW - Pain Measurement

KW - Risk Assessment

KW - Severity of Illness Index

KW - Time Factors

KW - Transplantation Conditioning

KW - Transplantation, Autologous

KW - Treatment Outcome

U2 - 10.3109/03009740903096622

DO - 10.3109/03009740903096622

M3 - SCORING: Journal article

C2 - 20132077

VL - 39

SP - 88

EP - 92

JO - SCAND J RHEUMATOL

JF - SCAND J RHEUMATOL

SN - 0300-9742

IS - 1

ER -