Therapies and medication

DMT received CR  PR  datestamped 

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: SMA
Record

DMT episode CR  PR    episode   

Related items in previous version: 15.34, 15.37, 15.38
DMT CR  PR 

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: SMA
Value ID Description
Ataluren

Ataluren (Translarna)

Casimersen

Casimersen (Amondys 45)

Eteplirsen

Eteplirsen (Exondys 51)

Golodirsen

Golodirsen (Vyondys 53)

Viltolarsen

Viltolarsen (Viltepso)

DMT status CR  PR 

Specifies whether this therapy is ongoing or stopped.

Item type: single selection
Consistency rules:

If a value for Stop date of this record is provided, this item must have the value Stopped.

Related items in previous version: 15.31
Usage in other datasets: SMA
Value ID Description
Ongoing

The individual has been receiving the therapy as of the date specified in Ongoing date and has been continuously receiving it from the date specified in Start date on (if specified)

Stopped

The individual has stopped continuously receiving the therapy since the date specified in Start date

DMT stopping reason CR  PR 

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
Value ID Description
Funding

Insurance coverage/funding

Availability

Availability

Side effects from drug

Side effects from the drug

Insufficient benefit

Insufficient benefit

Insufficient initial improvement

Insufficient initial improvement

Loss of response

Loss of response

Elective choice

Elective choice

Corticosteroid usage CR  PR  longitudinal 

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
Value ID Description
Currently

This is currently the case

Previously

This has previously been the case, but is not currently

Never

This has never been the case, neither previously nor currently

Sometime

This has been the case at some time, but it is unknown whether it is currently the case

Not currently

This is currently not the case, but it is unknown whether it has previously been the case

Record

Corticosteroid episode CR  PR    episode   

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
Corticosteroid drug CR  PR 

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)

Corticosteroid administration days on CR  PR 

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
Corticosteroid administration days off CR  PR 

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
Corticosteroid dosage CR  PR 

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
Corticosteroid stopping reason CR  PR 

Reason(s) for stopping this treatment.

Item type: multiple selection
Consistency rules:

Must not be provided in case Ongoing date is specified for this episode.

Related items in previous version: 15.15
Value ID Description
Side effects

Side effects

Elective choice

Elective choice

Insufficient benefit

Insufficient benefit

Cardiac treatment usage CR  PR  longitudinal 

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
Value ID Description
Currently

This is currently the case

Previously

This has previously been the case, but is not currently

Never

This has never been the case, neither previously nor currently

Sometime

This has been the case at some time, but it is unknown whether it is currently the case

Not currently

This is currently not the case, but it is unknown whether it has previously been the case

Record

Cardiac treatment episode CR      episode   

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 CR 

Cardiac treatment this episode refers to.

Item type: single selection
Consistency rules:

A value must not be specified for both this item and Other cardiac drug in the same episode.

Related items in previous version: 15.28
Usage in other datasets: LGMD
Value ID Description
Angiotensin receptor blockers

Angiotensin receptor blockers

ACE inhibitors

ACE inhibitors

Beta blockers

Beta blockers

Eplerenone

Eplerenone

Spironolactone

Spironolactone

Other diuretics

Diuretics other than eplerenone and spironolactone

Other cardiac drug CR 

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
Cardiac treatment stopping reason CR 

Reason(s) for stopping this treatment.

Item type: multiple selection
Consistency rules:

Must not be provided in case Ongoing date is specified for this episode.

Related items in previous version: 15.46
Value ID Description
Side effects

Side effects

Elective choice

Elective choice

Insufficient benefit

Insufficient benefit

Record

Allopathic drugs CR  PR      reference period 

Allopathic drug usage CR  PR 

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
Allopathic drugs CR  PR 

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
Value ID Description
Vitamin D

Vitamin D

Calcium

Calcium

Bisphosphonates

Bisphosphonates

Bone health

Drugs for bone health, not otherwise specified

Gastroesophageal reflux

Drugs for gastroesophageal reflux

Constipation

Drugs for constipation

Gastrointestinal

Gastrointestinal drugs, not otherwise specified

Antibiotics

Antibiotics

Influenza immunizations

Annual influenza immunizations

Pneumococcal immunizations

Annual pneumococcal immunizations

Respiratory

Respiratory drugs, not otherwise specified

Testosterone

Testosterone

Supplement

Supplement, not otherwise specified

Other allopathic drugs CR  PR 

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
Record

Allopathic drug episode CR  PR    episode   

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
Allopathic drug CR  PR 

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
Value ID Description
Vitamin D

Vitamin D

Calcium

Calcium

Bisphosphonates

Bisphosphonates

Bone health

Drugs for bone health, not otherwise specified

Gastroesophageal reflux

Drugs for gastroesophageal reflux

Constipation

Drugs for constipation

Gastrointestinal

Gastrointestinal drugs, not otherwise specified

Antibiotics

Antibiotics

Influenza immunizations

Annual influenza immunizations

Pneumococcal immunizations

Annual pneumococcal immunizations

Respiratory

Respiratory drugs, not otherwise specified

Testosterone

Testosterone

Supplement

Supplement, not otherwise specified

Other allopathic drug CR  PR 

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
Allopathic drug stopping reason CR  PR 

Reason(s) for stopping this treatment.

Item type: multiple selection
Consistency rules:

Must not be provided in case Ongoing date is specified for this episode.

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

Record

Rehabilitative interventions         reference period 

Rehabilitative interventions usage

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 Rehabilitative interventions has a value, this item must have the value Yes.

Related items in previous version: 15.44
Usage in other datasets: SMA
Rehabilitative interventions

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
Value ID Description
Physiotherapy

Physiotherapy sessions (e.g. stretches)

Respiratory physiotherapy

Respiratory physiotherapy sessions

Massage

Massage

Home programme

Home programme (e.g. stretches/exercises)

Hydrotherapy

Hydrotherapy/water-based activity

Contracture management using orthotics

Management of contractures using orthotics (e.g. ankle foot orthoses)

Spinal brace

Spinal brace

Occupational therapy

Occupational therapy sessions, or input/equipment for home

Speech therapy

Speech and language therapy sessions