Specifies whether the individual has ever received a disease-modifying therapy for their neuromuscular condition.
For SMA, these currently include nusinersen (Spinraza), Zolgensma (AVXS-101) and risdiplam (Evrysdi).
| Item type: | yes/no |
| Related items in previous version: | 11.00, 11.01 |
| Usage in other datasets: | DMD |
In version 2.1, this item was changed from longitudinal to datestamped.
This is an episode record: For each record instance, the following dates must be captured:
Start date: The month and year when the condition
described by the record started to hold, if known
Stop date: The month and year when the condition
ceased to hold, if applicable and known
Ongoing date: The month and year on which the
condition was known to hold, if applicable
See the introduction for more information.
The episode dates must be provided if and only if the therapy is administered over a period of time (currently nusinersen and risdiplam). For a therapy with a single administration (currently Zolgensma), the item DMT single administration date must be used. Registries should ensure that the individual's weight approximately at the beginning of the therapy is collected in the item Weight; in particular, if the therapy began before the baseline entry of the registration, the data collection form should include a field for the weight at therapy begin, which is then mapped to a value of Weight and datestamped with the therapy start date.
Note that the existence of an instance of this record implies that the individual is receiving or has received the therapy specified in DMT. It is not possible to express the fact that the individual has not received or is not receiving a certain therapy.
Specifies the disease-modifying therapy the individual is receiving or has received at some point.
| Item type: | single selection |
| Related items in previous version: | 11.02 |
| Usage in other datasets: | DMD |
| Value ID | Description |
|---|---|
| Nusinersen | Spinraza (nusinersen) |
| Onasemnogene abeparvovec | Zolgensma (AVXS-101) |
| Risdiplam | Evrysdi (risdiplam) |
Specifies whether this therapy is ongoing or stopped.
This item does not apply to therapies with a single administration (currently Zolgensma).
| Item type: | single selection |
| Consistency rules: | If a value for |
| Related items in previous version: | 11.00, 11.01 |
| Usage in other datasets: | DMD |
The disease-modifying therapies are collected in an episode record which already encodes whether the therapy is ongoing or has stopped by providing a value for Ongoing date or Stop date, respectively. However, in case the therapy is stopped, but the stop date is not known or has not been entered by the user and thus no value for Stop date can be provided, it is still important to encode this information. Therefore, unlike the other episode records in the dataset, the DMT record contains this item as a fallback.
Reason the individual has stopped receiving the therapy specified in DMT (in the case of a continuous treatment), or has received a different disease-modifying therapy after receiving a single-administration therapy specified in DMT.
In addition to the values listed for this item, registries may add further options in their data collection forms if relevant locally, and/or provide a free text field. However, additional options not specified in this dataset must not be provided in a data submission for a TREAT-NMD enquiry. Future revisions of this dataset may include additional values, and these can be suggested (along with justification) by using the “Report an issue with this item” function.
If Side effects from procedure or Side effects from drug is selected, a registry may remind the user to enter the side effects using the items in the group “Hospitalisations and comorbidities”. A registry may arrange their data collection form to collect side effects in any way that works best for them, for example in the same place as the disease-modifying therapies. However, the data structure must match the dataset items for hospitalisations and comorbidities to allow standardised data submission in case of enquiries.
| Item type: | single selection |
| Related items in previous version: | 11.05 |
| Usage in other datasets: | DMD |
In version 2.0, the option Lack of apparent benefit was renamed to Insufficient benefit and the options Scoliosis, Insufficient initial improvement and Loss of response were added.
Unit of the value specified in DMT dosage value.
| Item type: | single selection |
| Related items in previous version: | 11.06 |
Route of administration. If, for a specific therapy, only one value is possible, registries may omit this item from their forms and use a static value. The possible values will be amended in the future if additional therapies receive marketing authorisation.
| Item type: | single selection |
| Related items in previous version: | 11.08 |
| Value ID | Description |
|---|---|
| Intrathecal | Intrathecal injection |
| Intravenous | Intravenous |
| Oral | Oral or via feeding tube |
This item currently only applies to nusinersen. For any episodes where DMT is not nusinersen, this item is not intended for collection.
Comma-separated list of ISO 8601 durations that specify the intervals between the administrations. Repeating intervals may be specified as ISO 8601 recurring intervals by prepending Rn/, where n either is the number of repetitions, or absent in the case of unbounded repetitions.
For example, P14D, P14D, P30D, R/P4M specifies the (as of writing) current recommended dosing schedule for nusinersen: three loading doses administered at 14-day intervals, a fourth loading dose administered 30 days after the third dose, and maintenance doses once every 4 months thereafter.
| Item type: | restricted text |
| Related items in previous version: | 11.07 |
Because the recommended intervals between the administrations of nusinersen vary between 14 days and 4 months, the criterion for a deviation from the schedule as defined in the item DMT administration schedule deviation (a deviation of 14 days or more for any interval) may not be useful in every case. Therefore, the actual intervals of nusinersen administration may be collected in this item.
This item currently only applies to nusinersen and risdiplam. For any episodes where DMT is not nusinersen or risdiplam, this item is not intended for collection and thus not mandatory. However, this item may be applied to future disease-modifying therapies in subsequent versions of this dataset.
Deviation definition for nusinersen: Specifies whether the interval between any two consecutive administrations of nusinersen differs by 14 days or more from that specified in the applicable prescription information.
Deviation definition for risdiplam: Specifies whether the individual has ever failed to take the prescribed dosage of risdiplam for 7 consecutive days or more during this episode.
| Item type: | yes/no |
| Related items in previous version: | 11.09 |
This item currently only applies to nusinersen and risdiplam. For any episodes where DMT is not nusinersen or risdiplam, this item is not intended for collection and thus not mandatory. However, this item may be applied to future disease-modifying therapies in subsequent versions of this dataset.
Reason for not following the dosing schedule generally recommended at the time of administration. In case the value of DMT administration schedule deviation is Yes, this item must be collected. Otherwise, in particular when there was a schedule deviation which does not satisfy the condition specified in DMT administration schedule deviation, this item may also be collected.
In addition to the values listed for this item, registries may add further options in their data collection forms if relevant locally, and/or provide a free text field. However, additional options not specified in this dataset must not be provided in a data submission for a TREAT-NMD enquiry. Future revisions of this dataset may include additional values, and these can be suggested (along with justification) by using the “Report an issue with this item” function.
| Item type: | single selection |
| Related items in previous version: | 11.10 |
| Value ID | Description |
|---|---|
| Illness | Illness |
| Access problem | Access problem (e.g. funding, institutional/organisational issues) |
| Scoliosis surgery | Scoliosis surgery |
| Non-compliance | Non-compliance |
The value Other and the related free-text field were removed from the dataset in version 2.0. However, registries may include them in their data collection forms as noted above.
This item only applies to Zolgensma. For any episodes where DMT is not Onasemnogene abeparvovec, this item is not intended for collection and thus not mandatory.
Duration in days of the administration of prophylactic systemic corticosteroids in relation to this Zolgensma administration, excluding the final tapering period in which the dose is gradually reduced at the end of the administration.
| Item type: | integer |
| Unit: | days |
This item was added in version 2.0.
This item only applies to Zolgensma. For any episodes where DMT is not Onasemnogene abeparvovec, this item is not intended for collection and thus not mandatory.
Drug that was administered as prophylactic systemic corticosteroid in relation to this Zolgensma administration.
| Item type: | single selection |
| Value ID | Description |
|---|---|
| prednisone oral | prednisone (oral) |
| prednisolone oral | prednisolone (oral) |
This item was added in version 2.0.
For each anti-AAV9 antibody test, one record instance should be provided. This data should be collected for all patients, regardless of whether they have received Zolgensma or not.
These items were added in version 2.0.
Since version 2.1, this record is no longer longitudinal and the item Anti-AAV9 antibody test date was added instead. Furthermore, it was clarified that this data should be collected for all patients, regardless of whether they have received Zolgensma or not.
Antibody titre measured in this anti-AAV9 antibody test. Registries may collect the precise titre, but must be prepared to map the data to the two values provided here.
| Item type: | single selection |
| Value ID | Description |
|---|---|
| <= 1:50 | Antibody titre lower than or equal to 1:50 |
| > 1:50 | Antibody titre greater than 1:50 |
Days between this anti-AAV9 antibody test and the Zolgensma administration in relation to which it was performed. A positive value means that the test was performed before the Zolgensma administration, while a negative means that the test was performed afterwards. For example, a value of 1 denotes that the test was performed the day before the administration.
| Item type: | integer |
| Unit: | days |