Prevention of the progression of TMD to ML-DS by chemotherapeutical profylaxis can be possible.
ID
Bron
Verkorte titel
Aandoening
Transient myeloproliferative disorder in children with Down syndrome.
Ondersteuning
Stichting Sophia
Onderzoeksproduct en/of interventie
Uitkomstmaten
Primaire uitkomstmaten
1. To determine the population-based frequency of TMD;<br>
2. To establish and investigate the relation of TMD and ML-DS;<br>
3. To see if treatment of TMD can prevent TMD-associated mortality and the development of ML-DS in later life.
Achtergrond van het onderzoek
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.
Doel van het onderzoek
Prevention of the progression of TMD to ML-DS by chemotherapeutical profylaxis can be possible.
Onderzoeksopzet
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.
Onderzoeksproduct en/of interventie
Treatment will be advised when there is high MRD (above threshold) at week 8.
Publiek
Erasmus MC- Sophia Children's Hospital
POB 2060
M. Blink
Rotterdam 3000 CB
The Netherlands
+31 (0)10 7044640
m.blink@erasmusmc.nl
Wetenschappelijk
Erasmus MC- Sophia Children's Hospital
POB 2060
M. Blink
Rotterdam 3000 CB
The Netherlands
+31 (0)10 7044640
m.blink@erasmusmc.nl
Belangrijkste voorwaarden om deel te mogen nemen (Inclusiecriteria)
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.
Belangrijkste redenen om niet deel te kunnen nemen (Exclusiecriteria)
1. Children in whom the diagnosis Down syndrome can not be confirmed;
2. Complications that make sampling unwanted or impossible.
Opzet
Deelname
Opgevolgd door onderstaande (mogelijk meer actuele) registratie
Geen registraties gevonden.
Andere (mogelijk minder actuele) registraties in dit register
Geen registraties gevonden.
In overige registers
| Register | ID |
|---|---|
| NTR-new | NL1587 |
| NTR-old | NTR1667 |
| Ander register | METC ErasmusMC : 2007-168 |
| ISRCTN | ISRCTN wordt niet meer aangevraagd |