Treatment with hematopoietic stem cell transplantation or gene therapy is only beneficial in early disease stages of MLD. Determining which patients might benefit from treatment and which do not, can be difficult. Therefore, an international MLD treatment eligibility expert panel can be consulted. When there is a need to discuss a case, the panel is convened on an ad hoc basis.
- Cases eligible for discussion include patients with a confirmed diagnosis of MLD for whom possible benefits of treatment with hematopoietic stem cell transplantation or gene therapy are not straightforward. Also presymptomatic cases can be discussed .
- All physicians in Europe are encouraged to discuss these cases with this panel.
- Also physicians from other geographical areas are invited to share and discuss their cases.
Discuss a case
If you would like to discuss a case please contact the CPMS team and/or the MLD initiative.
- Physicians from EU countries can directly contact: email@example.com
- Physicians from non-EU countries should contact: MLDinitiative@amsterdamumc.nl
Before the meeting, the referring physician will be asked to complete a form with questions. Also, the patient should be informed and give informed consent. A digital meeting with international experts will be arranged. During the meeting, the referring physician briefly presents the case. A panel discussion will be held and an advice will be given. After the meeting, long-term follow-up will be ensured by including the patient in the MLDi registry.
A detailed description of the procedure can be found in the standard operating procedure on the MLD Treatment Eligibility Panel.
MLD treatment eligibility panel
The panel discussions are completely aligned with and part of the CPMS case discussions of the ERN-RND.
More information about online case discussions with CPMS can be found here: https://www.ern-rnd.eu/cpms/