Session Information
Session Type: Abstract Submissions
Session Time: 5:30PM-7:00PM
Thalidomide is an effective agent for several pediatric rheumatic diseases: Systemic onset Juvenile idiopathic arthritis(SOJIA), Behcet’s disease and recalcitrant skin disease in cSLE to name a few.
SOJIA is a common subtype of JIA seen in India. Patients with SOJIA are often steroid dependent and relapse on dose reduction. Tocilizumab though available is prohibitively expensive for the majority. Thalidomide also has a role to play for the therapy of recalcitrant oral aphthae in Behcet’s disease and can also be used for the calcinosis of Juvenile dermatomyositis.
Methods:
This study was a retrospective chart review of all children with pediatric rheumatic diseases who had been prescribed Thalidomide from 01/06/2009 to 15/01/2017.
Aims:
To study the usage, safety and efficacy of thalidomide in children with pediatric rheumatic diseases.
The Safety and side effects were studied for all patients(53/53). Efficacy was studied for patients with SOJIA(48/53).Systemic features of SOJIA recorded were: fever,rash,hepato and splenomegaly and lymphadenopathy.Articular features noted were the number of active joints.
Results:
Use: Thalidomide was taken by 53 children. SOJIA:48,SLE:1,Behcet’s disease:2,Juvenile dermatomyositis with calcinosis universalis:1,Muckle wells syndrome:1
Safety and Side effects:Thalidomide was very safe in all patients at a maximum daily dose of 5.64mg/kg/day. No NCV screening was done in children prior to starting thalidomide.
11/53 children(20.75%) reported minor side effects, 2 had constipation that necessitated dose reduction , 9 had increased somnolence. No neurological adverse events were noted. Thalidomide was used as a single bedtime dose to decrease somnolence. Maximum dose used was 200mg/day.
Efficacy in SOJIA patients: Thalidomide taken by 48/170(28%)patients with SOJIA followed at our unit. There were 18 girls and 30 boys.
Table 1
|
Median age at disease onset |
4.62yrs(0.7-13.5) |
|
|
Median age at disease diagnosis |
5.45yrs(1-13.75) |
|
|
Median delay to diagnosis |
4 months(1-48) |
|
|
Median age at thalidomide commencement: |
7yrs(1.7-17.25). |
|
|
Median duration of therapy with thalidomide |
16.5 months(1-93 months) |
|
|
Median dose of thalidomide |
2.56mg/kg/day(0.8-5.64mg/kg/day |
|
|
Median dose of oral steroids prior to thalidomide |
0.46mg/kg/day |
|
|
Median dose of oral steroids 6 months after thalidomide |
0.01mg/kg/day(0-1.18) |
P<0.001 |
|
Steroids stopped after Thalidomide |
28/48(58%) |
|
|
Median time taken for response in systemic features |
1 mth(0.5-18 months |
|
|
Median time taken to response for articular ds. |
3.5mths(1-15mths). |
|
|
Response in systemic features |
45/48(94%) |
|
|
Response in articular features |
24/48(50%) |
|
|
No response to Thalidomide |
3/48(6.2%) |
|
|
Longitudinal follow up |
||
|
No flare |
19/48(40%) |
|
|
Only articular flare |
17/48(35%) |
|
|
Both articular and systemic flare |
12/48(25%) |
|
|
Thalidomide stopped Disease remission No response/step up to Tocilizumab |
21/48(44%) 14/21(67%) 7/21(33%) |
|
Systemic flare was consequent to tapering/stopping Thalidomide. Articular flare was during ongoing Thalidomide therapy.
Conclusion:
1.Thalidomide is a safe drug with no neurological side effects noted in 53 children with a maximum daily dose of 5.64mg/kg/day over the maximum follow up period of 93 months.
2.It was used in 28% of total SOJIA patients followed at our unit
3.It was effective in 94% for systemic and in 50% for articular disease.
To cite this abstract in AMA style:
Agarwal M, Shivpuri A, Sawhney S. Use Of Thalidomide From A Tertiary Level Pediatric Rheumatology Centre In India [abstract]. Arthritis Rheumatol. 2017; 69 (suppl 4). https://acrabstracts.org/abstract/use-of-thalidomide-from-a-tertiary-level-pediatric-rheumatology-centre-in-india/. Accessed .« Back to 2017 Pediatric Rheumatology Symposium
ACR Meeting Abstracts - https://acrabstracts.org/abstract/use-of-thalidomide-from-a-tertiary-level-pediatric-rheumatology-centre-in-india/