Handgretinger, R

Holzer, U

Kuemmerle-Deschner, J

Lang, P

Mueller, I

Tzaribachev, N

Well, C

Pediatric Rheumatology

English

PubMed

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BioMed Central
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Pediatric Rheumatology
Open AccessPoster presentation
Successful autologous stem cell transplantation (ASCT) in a patient 
with juvenile dermatomyositis
U Holzer*, N Tzaribachev, J Kuemmerle-Deschner, C Well, P Lang, 
R Handgretinger and I Mueller
Address: Children's Hospital, Tuebingen, Germany
* Corresponding author    
Juvenile dermatomyositis (JDM) is a chronic inflamma-
tory disorder, which primarily affects muscle and skin.
Patients usually present with progressive muscle weakness
accompanied by an erythematous rash over the joints and
across the face. Articular, cardiac, pulmonary, and gas-
trointestinal manifestations may occur resulting in severe
morbidity.
We report a 16 year old patient, who was diagnosed with
JDM four years ago with severe muscle weakness and skin
involvement. Despite therapy including methotrexate,
steroids, immunoglobulins, cyclosporine A and rituximab
a sustained remission could not be achieved. The patient
developed progressive contractures due to persistent
inflammatory reactions. Due to treatment-refractory dis-
ease immunoablation followed by an autologous stem
cell transplantation (ASCT) was performed. The stem cells
were mobilized by application of 2 g/m2 cyclophospha-
mide. After immunoablation using ATG (thymoglob-
uline®) 10 mg/kg, cyclophosphamide 120 mg/kg and
fludarabine 150 mg/m2, CD3/CD19-depleted
CD34+stem cells (7.5 × 106/kg) including 2,9 × 104/kg T-
cells were transferred. The haematological reconstitution
with leucocytes >1000/ml and granulocytes >500/μl was
achieved on day +7 and day +8, respectively. Substitution
of erythrocytes or platelets was not necessary. No severe
infections or organ toxicity (WHO) were observed.
During the 9 months after this therapy a marked improve-
ment of the patient was observed clinically and in the
MRI. The Childhood Myositis Assessment Scale (CMAS)
changed from 6 to 43, the Karnofsky index increased from
50% to 90% post ASCT.
Taken together we could demonstrate for the first time
that an ASCT is a therapeutic option with low toxicity for
patients with severe, therapy-refractory JDM.
from 15th Paediatric Rheumatology European Society (PreS) Congress
London, UK. 14–17 September 2008
Published: 15 September 2008
Pediatric Rheumatology 2008, 6(Suppl 1):P221 doi:10.1186/1546-0096-6-S1-P221
<supplement> <title> <p>15<sup>th </sup>Paediatric Rheumatology European Society (PreS) Congress</p> </title> <editor>Wietse Kuis, Patricia Woo, Angelo Ravelli, Hermann Girschick, Michaël Hofer, Johannes Roth, Rotraud K Saurenmann, Alberto Martini, Pavla Dolezova, Janjaap van der Net, Pierre Quartier, Lucy Wedderburn and Jan Scott</editor> <note>Meeting abstracts – A single PDF containing all abstracts in this Supplement is available <a href="http://www.biomedcentral.com/content/files/PDF/1546-0096-6-S1-full.pdf">here</a>.</note> </supplement>
This abstract is available from: http://www.ped-rheum.com/content/6/S1/P221
© 2008 Holzer et al; licensee BioMed Central Ltd. 


Successful autologous stem cell transplantation (ASCT) in a patient with juvenile dermatomyositis

Successful autologous stem cell transplantation (ASCT) in a patient with juvenile dermatomyositis

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