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, Jahrgang 39, Nr. 1, 2010, S. 88-92.Publikationen: SCORING: Beitrag in Fachzeitschrift/Zeitung › SCORING: Zeitschriftenaufsatz › Forschung › Begutachtung
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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 -