No registrations found.
ID
Source
Brief title
Health condition
Transient myeloproliferative disorder in children with Down syndrome.
Sponsors and support
Stichting Sophia
Intervention
Outcome measures
Primary outcome
1. To determine the population-based frequency of TMD;
2. To establish and investigate the relation of TMD and ML-DS;
3. To see if treatment of TMD can prevent TMD-associated mortality and the development of ML-DS in later life.
Secondary outcome
1. To detect new genetic factors related to the progression of TMD to ML-DS;
2. To investigate the presence of lymphoid pre-leukemic clones in neonatal blood samples of TMD;
3. To describe the hematological abnormalities and clinical characteristics in patients with and without TMD.
Background summary
This is a prospective national screening study in which preferably all newborns with Down syndrome are screened for TMD.
If there is TMD, the children will be evaluated to see if there is a clinical indication for treatment.
Treatment is low-dose cytarabin.
Also, children who have high Minimal Residual Disease at week 8 will be treated.
Goal is to establish MRD-negativity (below threshold) at week 12.
All the children go into follow up to see if and when they develop ML-DS.
Study objective
Prevention of the progression of TMD to ML-DS by chemotherapeutical profylaxis can be possible.
Study design
1. Time point 1: < week 4;
2. Time point 2: at week 8;
3. Time point 2A: at week 10, only for those who were treated because of high MRD-levels at week 8;
4. Time point 3: at week 12.
Intervention
Treatment will be advised when there is high MRD (above threshold) at week 8.
Erasmus MC- Sophia Children's Hospital
POB 2060
M. Blink
Rotterdam 3000 CB
The Netherlands
+31 (0)10 7044640
m.blink@erasmusmc.nl
Erasmus MC- Sophia Children's Hospital
POB 2060
M. Blink
Rotterdam 3000 CB
The Netherlands
+31 (0)10 7044640
m.blink@erasmusmc.nl
Inclusion criteria
1. All children with Down syndrome;
2. Age < 4 weeks;
3. If blasts are present in pleural or pericardial effusion, or in a liverbiopsy
(in absence of blasts in peripheral blood);
4. Signed informed consent.
Exclusion criteria
1. Children in whom the diagnosis Down syndrome can not be confirmed;
2. Complications that make sampling unwanted or impossible.
Design
Recruitment
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| NTR-new | NL1587 |
| NTR-old | NTR1667 |
| Other | METC ErasmusMC : 2007-168 |
| ISRCTN | ISRCTN wordt niet meer aangevraagd |