Session Information
Date: Monday, October 22, 2018
Session Type: ACR Poster Session B
Session Time: 9:00AM-11:00AM
Background/Purpose:
Thalidomide is an effective agent for several pediatric rheumatic diseases: SOJIA, Behcet’s disease and recalcitrant skin disease in cSLE to name a few.
Systemic onset Juvenile idiopathic arthritis 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. Thalidomide also has a role to play for the therapy of recalcitrant oral aphthae in Behcet’s disease and is also used for the calcinosis of Juvenile dermatomyositis.This study was undertaken to study the usage, safety & efficacy of thalidomide in children with pediatric rheumatic diseases.
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 15/05/2018.
The safety & side effects were studied for all patients(71/71). Efficacy was studied for patients with SOJIA(65/71) due to small sample size of other diseases.
Results:
Use: Thalidomide was taken by 71 children. SOJIA:65,SLE:1,Behcet’s disease:3,Juvenile dermatomyositis with calcinosis universalis:1,Muckle wells syndrome:1
Safety and Side effects: Thalidomide was safe in all patients at a maximum daily dose of 5.7mg/kg/day. No NCV screening was done in children prior to starting thalidomide.
11/71 children(15.5%) 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 65/208(31%)patients with SOJIA followed at our unit. 23 girls and 42 boys.
|
Demographic Details |
range |
|
|
Median age at disease onset |
4.5years |
0.7-16 years |
|
Median age at disease diagnosis |
5.4 years |
1-16.16 years |
|
Median delay to diagnosis |
4 months |
1-48 months |
|
Median age at thalidomide commencement |
7 years |
1.7-18.08 years |
|
Median duration of therapy with thalidomide |
16 months |
1-110 months |
|
Median dose of thalidomide |
3.1 mg/kg |
1-5.7 mg/kg |
|
Median dose of oral steroids prior to thalidomide |
0.4mg/kg |
0-3.5mg/kg |
|
Median dose of oral steroids 6 months after thalidomide |
0.01mg/kg |
0-1.18mg/kg (P<0.001) |
|
Steroids stopped after 6 months of Thalidomide |
28/65 |
43% |
|
Response in systemic features |
60/65 |
92% |
|
Response in articular features |
37/65 |
57% |
|
No response to Thalidomide(systemic features/articular features) |
5/65 |
7.6% |
|
Median time taken for response in systemic features |
1 month |
0.5-18 months |
|
Median time taken to response for articular ds. |
4 months |
1-16 months |
|
Longitudinal follow up |
||
|
No flare ever |
30/65=46% |
|
|
Only articular flare |
13 |
|
|
Both articular and systemic flare |
17 |
|
|
Thalidomide stopped |
37/65=57% |
|
|
No response/step up to Tocilizumab |
5/37=13.5% |
|
|
Disease remission |
17/37=46% |
|
|
Persistent articular disease not responding to Thalidomide after 6 months |
15/37=40.5% |
|
Conclusion:
1.Thalidomide is a safe drug with no neurological side effects noted in 71 children with a maximum daily dose of 5.7mg/kg/day over the maximum follow up period of 110 months.
2.It was used in 1/3 of total SOJIA patients
3.It was effective in 92% for systemic and in 57% for articular disease.
To cite this abstract in AMA style:
Agarwal M, Sawhney S. Use,Safety and Efficacy of Thalidomide from a Tertiary Level Pediatric Rheumatology Centre in India [abstract]. Arthritis Rheumatol. 2018; 70 (suppl 9). https://acrabstracts.org/abstract/usesafety-and-efficacy-of-thalidomide-from-a-tertiary-level-pediatric-rheumatology-centre-in-india/. Accessed .ACR Meeting Abstracts - https://acrabstracts.org/abstract/usesafety-and-efficacy-of-thalidomide-from-a-tertiary-level-pediatric-rheumatology-centre-in-india/