N. Wulffraat et al., Autologous haemopoietic stem-cell transplantation in four patients with refractory juvenile chronic arthritis, LANCET, 353(9152), 1999, pp. 550-553
Citations number
27
Categorie Soggetti
General & Internal Medicine","Medical Research General Topics
Background Autologous haemopoietic stem-cell transplantation (AHSCT) had be
en described as a possible treatment for severe autoimmune disease refracto
ry to conventional treatment. We report the first four children with severe
forms of juvenile chronic arthritis (JCA) treated with AHSCT.
Methods We studied three children with systemic JCA and one child with poly
articular JCA. Unprimed bane marrow was taken 1 month before AHSCT. T-cell
depletion of the graft was done with CD2 and CD3 antibodies. We used a prep
arative regimen of antithymocyte globulin (20 mg/kg), cyclophosphamide (200
mg/kg) and low-dose total body irradiation (4 Gy). Methotrexate and cyclos
porin were stopped before AHCST, prednisone was tapered after 2 months.
Findings Our patients showed a drug-free follow-up of 6-18 months with a ma
rked decrease in joint swelling, pain, and morning stiffness. Erythocyte se
dimentation rate, C-reactive protein, and haemoglobin returned to almost no
rmal values within 6 weeks. Despite T-cell depletion there was a rapid immu
ne reconstitution in three out of four children. Two patients developed a l
imited varicella tester virus eruption, which was treated by aciclovir.
Interpretation AHSCT for severe JCA was well tolerated and induced a remiss
ion of disease in four children with JCA that was resistant to conventional
treatment. Prolonged prednisane-free growth catch-up and general well-bein
g is a major therapeutic gain in such children. The actual follow-up is too
short, however, for us to conclude that these children are completely cure
d of their disease.