Stem Cell Transplants for JRA

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Stem-Cell Transplant Effective in Some Children with JRA

Paper: Autologous haemopoietic stem-cell

transplantation in patients with

refractory juvenile chronic arthritis.

Authors: Wulffraat N, van Royen A, Bierings M, et

al.

Ref: Lancet 1999;353:550-53.

Type: Preliminary Case Reports

Summary: Autologous haemopoietic stem-cell

transplantation

(AHSCT) has been proposed as a potential

treatment for

patients with refractory severe autoimmune

diseases such as

systemic sclerosis, rheumatoid arthritis,

multiple sclerosis, and

SLE. A small proportion of children with

juvenile rheumatoid

arthritis (JRA) are refractory to treatment

with traditional

therapies including NSAIDs and

immunosuppressive drugs.

These children may benefit from more

aggressive therapy

that suppresses the autoreactive T-cellular

immunity and

gives a normal immune competence by the

infusion of na•ve

T- cell precursors. This report describes the

follow-up for up

to 18 months in four children with severe and

longstanding

JRA treated with AHSCT.

Unprimed bone marrow was taken from each child

(ages 6, 7

and 11) one month before the procedure. T-cell

depletion of

the graft was accomplished using CD2 and CD3

antibodies.

Methotrexate and cyclosporine were stopped

before AHCST

and prednisone was tapered after the first 2

months.

These four patients continue in a drug-free

follow-up of

between 6 and 18 months with a marked decrease

in joint

swelling, pain and morning stiffness. The

erythrocyte

sedimentation rate, C-reactive protein

concentration, and

hemoglobin concentration returned to normal

within 6 weeks

of the procedure. Despite the T-cell

depletion, there was a

rapid renewal of the immune response in three

of the four

children. Two children developed a limited

varicella zoster

eruption treated withj acyclovir. Improvements

in daily

functioning that resulted from physiologic

improvements

associated with the tapering of

corticosteroids were

observed.

These preliminary findings suggest that AHSCT

was well

tolerated and induced an effective remission

in the four

children with JRA who had been resistant to

conventional

treatments. Other benefits may also derive

from this therapy

including a prolonged prednisone-free period

during which

growth suppression will not be a problem.

While the

18-month follow-up period is still relatively

short to conclude

that the patients have been cured by this

procedure, the data

provide strong evidence for its clinical

benefits in selected

patients.