Efficacy of early treatment of severe juvenile dermatomyositis with intravenous methylprednisolone and methotrexate.

A pilot study was conducted to assess the efficacy of early treatment of severe juvenile dermatomyositis (JDMS) patients with intravenous methylprednisolone (IVMP) and methotrexate (MTX). Twelve children diagnosed with severe JDMS were treated with IVMP and MTX. Six patients were treated early (within 6 weeks of the diagnosis) while in the other six patients, MTX was started 5-72 months after the diagnosis was made. The clinical responses of the patients to treatment, including alterations in muscle strength, muscle enzyme levels and corticosteroid dosage as well as the development of side-effects, were recorded. The indications for starting the treatment were defined and documented. The primary measures of response were resolution of the clinical indications for treatment, decreased activity of the disease manifestations and tapering of the corticosteroids to the minimal dose or discontinuation without clinical or biochemical flare. The main indications for starting IVMP and MTX were dysphagia and severe cutaneous vasculitis. All the patients received MTX orally for at least 8 months, as well as IVMP (30 mg/kg/dose), but none of the patients was on additional second-line treatments. The six patients who were treated early with MTX showed a significant clinical improvement. In five out of the six, the corticosteroid dosage was eventually reduced to <5 mg/day. None of them developed calcinosis. In contrast, two of the six patients who were treated late with MTX developed calcinosis. This study suggests that MTX and IVMP are a useful combination in the early treatment of severe JDMS. Given the fact that our sample was small, further studies in a controlled trial are necessary to confirm these findings.

Methotrexate therapy in systemic-onset juvenile rheumatoid arthritis in Saudi Arabia: a retrospective analysis.

Eighteen patients (nine girls, nine boys) with systemic onset juvenile rheumatoid arthritis (SO-JRA) treated with methotrexate (MTX) for a mean period of 18 months (range 6-41 months) were analysed to evaluate the safety and efficacy of MTX in this disease subtype. The MTX dose ranged from 2.5 to 15 mg/week with a mean cumulative dose of 684.9 mg/patient at the last follow-up visit. Systemic features were severe in 10 patients before MTX was started. None of these patients showed systemic features at the last follow-up visit. Sixteen patients (89%) showed improvement in both the active joint count (from a mean of 12.0 to 1.3 joints/patient) and function class (from a mean of 3.0 to 1.3) while receiving MTX. Eleven patients (61%) showed a significant decrease in the erythrocyte sedimentation rate (>50% of the initial value), an improvement in anaemia (haemoglobin >2 g) and reduced thrombocytosis (platelets 2 x 10[5]). Of the patients receiving corticosteroids, three patients (20%) were able to discontinue prednisone and the dose was reduced to less than 50% of the initial dose in seven patients (47%). At these doses of MTX, no gastrointestinal, hepatic or haematological toxicity was encountered and none of the patients withdrew because of toxicity or lack of efficacy. This report suggests that MTX is an effective and safe treatment in controlling systemic and articular features in this subtype of JRA.

Juvenile systemic lupus erythematosus in 60 Saudi children.

A ten-year retrospective analysis of the clinical features and survival of 60 Saudi children with systemic lupus erythematosus (SLE) was made. All the patients fulfilled the 1982 American College of Rheumatologyâs revised criteria for SLE and had had the disease at or before the age of 16 years. The female to male ratio was 5:1, the mean age of onset was 12.1 years (range 1.6-16 years), and the mean duration of follow-up was 4.7 years (range 2.2-11). Thirty-eight patients (63%) were diagnosed correctly before referral to KFSH&RC or KKUH. The mode of presentation was as follows: 55 patients had musculoskeletal involvement (91.6%), 49 patients had skin involvement (81.6%), 40 patients had hematological abnormalities (66.6%), 39 patients had renal disease (65%), 10 patients had pulmonary involvement (16%), 23 patients had cardiovascular disease (38%) and 18 patients had central nervous system involvement. During the study period four patients died (6.6%)âtwo of renal failure, one from meningitis and one from severe sepsis. This is the largest collection of childhood systemic lupus erythematosus from the Middle East and it shows that SLE is more common in Saudis than was hitherto believed, and that it has a high rate of organ involvement.

Juvenile Behçet's disease in Arab children.

Behçet's disease is a chronic, relapsing, multisystem disease characterized by the clinical triad of genital ulcers, oral ulcers and ocular involvement. Twelve Saudi children are presented, all of whom satisfied the international criteria for the classification of Behçet's disease and whose initial manifestations appeared at or before the age of 16 years. The male-to-female ratio was 1.4:1. The mean age at onset was 11.5 years (range 7-16 years) and the mean duration of disease was 6.5 years (range 3-13 years). Oral ulcers were present in all patients (100%), genital ulcers in 11 patients (91%), ocular involvement in the form of anterior and/or posterior uveitis in 6 patients (50%), skin manifestations in 10 patients (83%), musculoskeletal symptoms in 9 patients (75%), and central nervous system involvement in 6 patients (50%). One patient had thrombophlebitis and another had pulmonary artery aneurysm. No renal, cardiovascular or gastrointestinal abnormalities were detected. The pathergy test was positive in 3/7 patients. HLA B5 (W51) typing was positive in 5/10 patients. This report of juvenile Behçet's disease in Saudi children suggests that this multisystem disease has an aggressive nature and should be considered in the differential diagnosis of childhood vasculitis in endemic areas.

Syndrome of camptodactyly, arthropathy and coxa vara (CAC syndrome).

Three Saudi children (two female, one male) are described who presented with familial arthropathy associated with congenital camptodactyly. This rare but recognized clinical entity has a variable clinical presentation and may be associated with pericarditis and coxa vara. Camptodactyly was observed in the neonatal period in all patients, while joint swelling was observed between the third and 11th month. Pericarditis was suspected in the referral hospital in one patient but was not subsequently confirmed at our institution, raising the possibility that pericarditis may be reversible. Radiological examination of the hips showed coxa vara with short femoral neck in all patients. Synovial biopsy in the three patients revealed proliferating synovial epithelium with moderate fibrocollagenous densities and multinucleated giant cells, occasional lymphocytes or neutrophils but no plasma cells were identified. This is the first series of this familial arthropathy with a triad of camptodactyly, arthropathy and coxa vara (CAC syndrome) in Saudi Arabia which is to be considered in patients where more than one family member has juvenile arthritis.

Radiological features in congenital camptodactyly, familial arthropathy and coxa vara syndrome.

The association of congenital camptodactyly, familial arthropathy and coxa vara is a rare but recognized clinical entity. The radiological manifestations were reviewed in five patients. In the hips, coxa vara, short broad femoral necks and intraosseous cysts were demonstrated in all patients. Abnormal modeling of the acetabulum, increased joint space, effusion, small iliac wings and intraosseous herniation of fluid were found in four out of five patients. Somewhat flat, slightly irregular femoral heads were seen in three patients. In the knees, effusion was demonstrated in all five patients and thick cartilage in three. The elbow, wrist and ankle joints showed abnormal modeling with evidence or suspicion of intraarticular fluid in the majority of patients. Flexion deformities of the fingers were seen in all patients.