Specifies whether the individual has ever received a disease-modifying therapy for their neuromuscular condition.
For DMD, these currently include the therapies listed as possible values for the item DMT below. Registries should ensure that the corresponding question in their data collection forms applies to all DMTs, even when further therapies are approved in the future. In case the question references specific therapies (e.g. "Have you received any of the following therapies?"), this means that the list should always be kept up to date (and if required, an ethics approval should cover such updates). Alternatively, the question can be worded in a way that does not rely on an exhaustive list of therapies (e.g. "Have you ever received a disease-modifying therapy?" together with an explanation and a non-exhaustive list of currently approved therapies).
| Item type: | yes/no |
| Related items in previous version: | 15.31 |
| Usage in other datasets: | sNMD |
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.
| Related items in previous version: | 15.34, 15.37, 15.38 |
| Usage in other datasets: | sNMD |
Specifies the disease-modifying therapy the individual is receiving or has received at some point.
When a new therapy is approved, this list will be updated in the dataset. However, there may be a time lag between the approval and the dataset update. Therefore, registries should either update their data collection forms as soon as possible after a relevant new therapy is approved without waiting for the addition to the dataset, or add a free-text field that can be used to specify any new therapy. As soon as the new value is added to the dataset, any entries containing a new therapy should be validated and mapped accordingly.
| Item type: | single selection |
| Related items in previous version: | 15.32, 15.35 |
| Usage in other datasets: | sNMD |
| Value ID | Description |
|---|---|
| Ataluren | Ataluren (Translarna) |
| Casimersen | Casimersen (Amondys 45) |
| Eteplirsen | Eteplirsen (Exondys 51) |
| Golodirsen | Golodirsen (Vyondys 53) |
| Viltolarsen | Viltolarsen (Viltepso) |
Specifies whether this therapy is ongoing or stopped.
| Item type: | single selection |
| Consistency rules: | If a value for |
| Related items in previous version: | 15.31 |
| Usage in other datasets: | sNMD |
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.
| Item type: | single selection |
| Related items in previous version: | 15.39 |
| Usage in other datasets: | SMA |
Currently, Previously and
Never with suitable user-friendly wording. See the
introduction
for more information and alternative ways to collect this item.
Specifies whether the individual is currently taking or has previously taken corticosteroids for their neuromuscular condition.
| Item type: | single selection |
| Related items in previous version: | 15.01 |
| Usage in other datasets: | LGMD |
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.
At baseline, details for the most recent or ongoing corticosteroid usage must be given in an instance of this record. At registry updates, a previously ongoing episode should be updated and any further episodes added as additional instances.
The stop date should be the date of the last administration during this episode.
| Related items in previous version: | 15.07, 15.13, 15.14 |
Drug taken during this episode, excluding the tapering period. In case the drug is changed during one treatment, multiple episodes must be provided.
| Item type: | single selection |
| Related items in previous version: | 15.02, 15.08 |
| Value ID | Description |
|---|---|
| prednisone oral | prednisone (oral) |
| prednisolone oral | prednisolone (oral) |
| deflazacort oral | deflazacort (oral) |
Integer item specifying the number of days in a row the individual has been taking the corticosteroid. In combination with the subsequent item Corticosteroid administration days off, it specifies the administration schedule. The following table shows examples for various administration frequencies, where the column "Days on" contains the value of this item and "Days off" contains the value of the item Corticosteroid administration days off:
| Administration frequency | Days on | Days off |
|---|---|---|
| Daily | 1 | 0 |
| Every other day | 1 | 1 |
| Weekend dosing | 2 | 5 |
| 10 days on/10 days off | 10 | 10 |
| 20 days on/10 days off | 20 | 10 |
| etc. |
For daily administration, value of Corticosteroid administration days on must be 1. Registries should offer a predefined list of options for the most common administration frequencies (for example those shown in the table above) which are automatically mapped to the according values of the items Corticosteroid administration days on and Corticosteroid administration days off, while displaying number input fields only to cover further cases.
| Item type: | integer |
| Related items in previous version: | 15.04, 15.05, 15.10, 15.11 |
| Usage in other datasets: | LGMD |
Number of days in a row the individual has not been taking the corticosteroid. See the description of Corticosteroid administration days on for examples.
| Item type: | integer |
| Related items in previous version: | 15.04, 15.05, 15.10, 15.11 |
| Usage in other datasets: | LGMD |
Daily dosage of the corticosteroid, relative to the individual's body weight. If the corticosteroid was not administered daily, the dosage given on the administration days must be specified. For example, if the individual took 2 mg/kg on two days of the week and nothing on the other days, then 2 must be provided as value.
| Item type: | decimal |
| Unit: | milligrams per kilogram (mg/kg) of body weight |
| Related items in previous version: | 15.06, 15.12 |
Reason(s) for stopping this treatment.
| Item type: | multiple selection |
| Consistency rules: | Must not be provided in case |
| Related items in previous version: | 15.15 |
| Value ID | Description |
|---|---|
| Side effects | Side effects |
| Elective choice | Elective choice |
| Insufficient benefit | Insufficient benefit |
Currently, Previously and
Never with suitable user-friendly wording. See the
introduction
for more information and alternative ways to collect this item.
Specifies whether the individual is currently receiving or has previously received cardiac treatment.
| Item type: | single selection |
| Related items in previous version: | 15.27 |
| Usage in other datasets: | LGMD |
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.
At baseline, details for all cardiac treatments in the last 12 months should be given in instances of this record. At registry updates, any previously ongoing episodes should be updated and any further episodes added as additional instances.
| Related items in previous version: | 15.28 |
Cardiac treatment this episode refers to.
| Item type: | single selection |
| Consistency rules: | A value must not be specified for both this item and |
| Related items in previous version: | 15.28 |
| Usage in other datasets: | LGMD |
International Nonproprietary Name (INN) of a cardiac drug that this episode refers to but which is not listed as a possible value of the item Cardiac treatment.
| Item type: | free text |
| Related items in previous version: | 15.29 |
| Usage in other datasets: | LGMD |
Reason(s) for stopping this treatment.
| Item type: | multiple selection |
| Consistency rules: | Must not be provided in case |
| Related items in previous version: | 15.46 |
| Value ID | Description |
|---|---|
| Side effects | Side effects |
| Elective choice | Elective choice |
| Insufficient benefit | Insufficient benefit |
This is a reference period record: For each record instance, the following dates (consisting of month and year) must be captured:
Begin date specifies the begin of the period to which
the question refers
End date specifies the end of the period to which the
question refers
At baseline, the period should range over the last 12 months before the date of entry; at follow-ups, it should range from the last update to the date of entry. See the introduction for more information.
Specifies whether the individual has taken any prescribed allopathic drugs (to manage symptoms - not disease-modifying) or supplements during this reference period. If the value is Yes, details for each drug may be specified in instances of the record Allopathic drug episode.
This item does not apply to the usage of corticosteroids as a treatment for the neuromuscular condition or to the usage of cardiac drugs. These usages are specified in the items Corticosteroid usage and Cardiac treatment, respectively.
Only the usage of drugs or supplements with a duration of 14 or more consecutive days should be considered (apart from immunizations).
| Item type: | yes/no |
| Related items in previous version: | 15.17, 15.41 |
| Usage in other datasets: | SMA |
Prescribed allopathic drugs or supplements the individual has taken during some time in the period from Begin date to End date.
Please note that the inclusion of a drug or supplement in this list does not indicate TREAT-NMD endorsement.
The four options containing "not otherwise specified" in the description (Bone health, Gastrointestinal, Respiratory and Supplement) should only be used in case no more specific information is available. For example, if a registry form contains the question "Have you taken any drugs for bone health?" and the reply is "Yes", but no further details are known, then the value would be Bone health. But if it is known that the individual has taken bisphosphonates, then only Bisphosphonates (and not Bone health) should be provided as value of this item.
Only the usage of drugs or supplements with a duration of 14 or more consecutive days should be considered (apart from immunizations).
| Item type: | multiple selection |
| Related items in previous version: | 15.18, 15.19, 15.21, 15.22, 15.24 |
| Usage in other datasets: | SMA |
Comma-separated list of the International Nonproprietary Names (INNs) of other drugs.
Corticosteroids taken as a treatment for the neuromuscular condition as well as cardiac drugs must not be specified here. Instead, the items Corticosteroid drug and Cardiac treatment (or Other cardiac drug) must be used instead.
| Item type: | free text |
| Related items in previous version: | 15.20, 15.22, 15.25, 15.30 |
| Usage in other datasets: | SMA |
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.
Details of the allopathic drugs or supplements the individual has taken.
The usage of corticosteroids taken as a treatment for the neuromuscular condition as well as the usage of cardiac drugs must not be specified here. Instead, the episode records Corticosteroid episode and Cardiac treatment must be used instead.
See the item Allopathic drugs for information regarding the options containing "not otherwise specified". Registries may include a free-text field to collect the names of further supplements not listed here.
Only the usage of drugs or supplements with a duration of 14 or more consecutive days should be considered (apart from immunizations).
| Related items in previous version: | 15.19, 15.22, 15.25 |
Prescribed allopathic drug or supplement the individual has taken.
Please note that the inclusion of a drug or supplement in this list does not indicate TREAT-NMD endorsement.
| Item type: | single selection |
| Related items in previous version: | 15.19, 15.21, 15.22, 15.24 |
| Usage in other datasets: | SMA |
International Nonproprietary Name (INN) of the other drug taken.
| Item type: | free text |
| Related items in previous version: | 15.20, 15.22, 15.25, 15.30 |
| Usage in other datasets: | SMA |
Reason(s) for stopping this treatment.
| Item type: | multiple selection |
| Consistency rules: | Must not be provided in case |
| Related items in previous version: | 15.19, 15.22, 15.25 |
| Value ID | Description |
|---|---|
| Side effects | Side effects |
| Elective choice | Elective choice |
| Insufficient benefit | Insufficient benefit |
This is a reference period record: For each record instance, the following dates (consisting of month and year) must be captured:
Begin date specifies the begin of the period to which
the question refers
End date specifies the end of the period to which the
question refers
At baseline, the period should range over the last 12 months before the date of entry; at follow-ups, it should range from the last update to the date of entry. See the introduction for more information.
Specifies whether the individual has received any rehabilitative interventions in this period.
For the purposes of this dataset, “rehabilitative interventions” refers to the management of the individual’s condition and quality of life through therapies from allied health professionals such as physiotherapists and speech therapists.
| Item type: | yes/no |
| Consistency rules: | If |
| Related items in previous version: | 15.44 |
| Usage in other datasets: | SMA |
Rehabilitative interventions the individual has received at some time during the period from Begin date to End date.
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: | multiple selection |
| Related items in previous version: | 15.44 |
| Usage in other datasets: | SMA |