Treatment eligibility panel

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.

When is discussion and advice by this panel recommended?

  • 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.

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.

Please contact us if you face an MLD patient for whom possible benefits of treatment are not straightforward

More information

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:

Are you a physician and do you want advice about an MLD patient?

Contact us!