Q23 – 63: Clinical Status Prior to Preparative Regimen

*Testing Prior to the Start of the Preparative Regimen / Infusion:
Report all findings within two months prior to the start of the preparative regimen / infusion for the Clinical Status Prior to Preparative Regimen section. If an assessment was performed multiple times, report the most recent results.

*Enzyme activity / enzyme substrate testing:
Do not include the recipient’s results of enzyme activity / enzyme substrate testing performed at diagnosis.

Questions 23 – 24: Was enzyme activity and/or enzyme substrate tested?

Indicate if the recipient was tested for enzyme activity and / or enzyme substrate prior to the start of the preparative regimen / infusion. The type of testing performed is specific to leukodystrophy subtype. Types of testing can include enzyme assays, urine testing, etc.

If the recipient’s enzyme activity and / or enzyme substrate was tested, select Yes and report the date of testing. If testing is performed multiple times, report the most recent date and results prior to the start of the preparative regimen / infusion.

If the exact date is not known, use the process described in General Instructions, Guidelines for Completing Forms.

If the enzyme activity and / or enzyme substrate was not tested prior to the start of the preparative regimen / infusion or it is unknown if testing occurred, select No or Unknown, respectively and continue with question 27.

Questions 25 – 26: Recipient result

Indicate whether the results of the recipient’s enzyme activity and / or enzyme substrate testing were Normal or Abnormal and if documentation was submitted to CIBMTR (CIBMTR recommends attaching the enzyme / storage activity testing).

For further instructions on how to attach documents in FormsNet3SM, refer to the Training Guide.

Questions 27 – 28: Was the total neurologic function scale (NFS) score obtained? (ALD recipients only)

The total neurologic function scale (NFS) is a 25-point scale used to assess severity of neurological dysfunction and is done by scoring 15 disabilities.

Indicate Yes or No if a NFS score was obtained prior to the preparative regimen / infusion. This information will be documented within a physician’s note.

If a total NFS score was obtained, select Yes, and report the date of assessment. If the total NFS was obtained multiple times prior to the preparative regimen / infusion, report the date and results of the most recent assessment.

If the exact date is not known, use the process described in General Instructions, Guidelines for Completing Forms.

If the total NFS score was not obtained prior to the preparative regimen / infusion or it is not known if a score was obtained, select No, and continue with question 45.

*Total Neurologic Function Scale (NFS) Score and Domain Clinical Scores
When reporting the total NFS and domain clinical scores, report the scores based on documentation within the physician’s progress note. If the total NFS score is documented but the domain clinical scores are not provided, only report the total NFS score and leave the data fields for the domain clinical scores blank. Similarly, if only the domain clinical scores are known, report the domain clinical scores and leave the total NFS score blank.

Question 29: Specify total neurologic function scale score

Report the total neurologic function scale (NFS) score as documented by the physician. The total NFS score will be a value between 1-25.

If the NFS score is not known and only the domain clinical scores are documented, leave this data field blank.

Questions 30 – 44: Select known domain clinical score(s) (check all that apply)

Select the known domain clinical score(s) and report the score(s) as documented by the physician.

• Hearing / auditory processing problems: Trouble with hearing.
• Aphasia / apraxia: Speech disorder / impairment.
• Loss of communication: Loss of the ability to communicate.
• Vision impairment / fields cut: Reduced field of vision.
• Cortical blindness: Total or partial loss of vision due to damage to the occipital cortex.
• Swallowing difficulty or other central nervous system dysfunction: Dysphagia due to damage of the nervous system.
• Tube feeding: Percutaneous endoscopic gastrostomy (PEG), esophagogastroduodenoscopy (EGD), or G-tube insertion.
• Running difficulties / hyperreflexia: Muscles are less responsive to stimuli. Causing trouble holding, running, driving etc.
• Walking difficulties / spasticity / spastic gait (no assistance): Trouble walking, and / or abnormal muscle tightness due to prolonged muscle contractions.
• Spastic gait (needs assistance) wheelchair required: Stiff, often foot dragging walk, due to prolonged muscle contractions on one side. Assistance with wheelchair required.
• No voluntary movement: Absence of voluntary movements such as moving fingers, toes, sitting upright, etc.
• Episodes or urinary or fecal incontinency: Loss of bladder and / or bowel control with episodes of any urinary or bowel incontinence.
• Total urinary or fecal incontinency: Total loss of bladder and / or bowel control. This will require continuous use of catheter.
• Nonfebrile seizures: A single, uncontrolled electrical activity in the brain, which may produce a physical convulsion, minor physical signs, thought disturbances or a combination of symptoms. Nonfebrile seizures are characterized as spontaneous recurrent seizures unrelated to fever.

If only the total NFS score is known and not the domain clinical scores, leave these questions blank.

Question 45: Is there a history of seizures attributed to the underlying disease at any time prior to the preparative regimen?

Indicate Yes if there was a history of seizures attributed to the underlying disease at any time prior to the preparative regimen / infusion. If there was no history of seizures attributed to the underlying disease or it is not known, indicate No, and continue with question 47.

Question 46: Were any of the seizures considered nonfebrile?

Report Yes if any of the seizures prior to the preparative regimen / infusion were considered nonfebrile (spontaneous recurrent seizures unrelated to fever). If all seizures prior to the start of the preparative regimen / infusion were febrile, select No.

Questions 47 – 48: Was cerebrospinal fluid (CSF) testing done prior to the preparative regimen?

Indicate if cerebrospinal fluid (CSF) testing was completed prior to the preparative regimen / infusion. CSF is collected via lumbar puncture or spinal tab.

If testing was performed, select Yes and report the date of the most recent CSF testing.

If the exact date is not known, use the process described in General Instructions, Guidelines for Completing Forms.

If CSF testing was not performed prior to the preparative regimen / infusion or it is not known, select No or Unknown, respectively and continue with question 52.

Questions 49 – 51: Specify known CSF result(s) (check all that apply)

Report the known CSF results.

• Opening Pressure: Opening pressure is measured during the lumbar puncture; this is a measurement of intracranial pressure. If the opening pressure is known, select this option and report the opening pressure value in question 50.
• Total Protein: Total protein is a measurement used to determine the levels of protein in cerebrospinal fluid. If the total protein is known, select this option and report the CSF total protein value in question 51.

In the rare case where neither the opening pressure or the total protein was known, leave question 49 blank, override the validation error using the code “unknown,” and continue with question 52.

Question 52: Date of most recent MRI prior to the preparative regimen

Magnetic resonance imaging (MRI) is an imaging technique used to form pictures of the anatomy and the physiological processes of the body. MRI are used to assess recipient’s with leukodystrophy.

Report the date of the most recent MRI performed prior to the preparative regimen / infusion.

If the exact date is not known, use the process described in General Instructions, Guidelines for Completing Forms.

Question 53: Specify MRI results

Specify the results of the most recent MRI performed prior to the start of the preparative regimen / infusion. Indicate if the results were Normal or Abnormal as determined by the radiologist or physician. If results were Normal, continue with question 56.

Questions 54 – 55: Was gadolinium contrast used for this assessment?

Gadolinium contrast is often used in MRI assessments to enhance imagining, improving the visibility of inflammation, blood vessels and blood supply.

If gadolinium contrast was used, select Yes in question 54. If gadolinium enhancement was reported, select Yes in question 55. If gadolinium contrast was used, gadolinium enhancement will be noted in the MRI report and can be suggestive of abnormalities.

If gadolinium contrast was not used in the MRI assessment, select No and continue with question 56.

Question 56: Is a copy of the MRI report attached? (CIBMTR recommends attaching the MRI report)

Report Yes or No if a copy of the MRI report is attached. For instructions on how to attach documents in FormsNet3SM, refer to the Training Guide.

Questions 57 – 58: Were nerve conduction velocities tested at any time prior to the preparative regimen?

Nerve conduction velocity (NCV) testing measures how quickly an electrical impulse moves through the nerve and can identify nerve damage. This is procedure is typically performed by a neurologist.

Indicate if NCV testing was performed at any time prior to the preparative regimen / infusion. If Yes, report the date of the most recent testing. If NCV testing was not done or it is unknown, report No or Unknown, respectively and continue with question 61.

Questions 59 – 60: Specify Results

Report whether the results of the recipient’s NCV testing was Normal or Abnormal. Additionally, indicate if documentation was submitted to CIBMTR (CIBMTR recommends attaching the nerve conduction velocities tests).

The results of the NCV test may be found in the procedure / results report or in the neurologist’s note.

For instructions on how to attach documents in FormsNet3SM, refer to the Training Guide.

Questions 61 – 63: Was a neurocognitive test administered at any time prior to the preparative regimen?

A neurocognitive test is an assessment completed by a neuropsychologist, used to assess the cognitive function of a recipient. Indicate if a neurocognitive test was administered at any time prior to the preparative regimen / infusion. This information will be found within a progress note by the neuropsychologist.

If neurocognitive testing was performed, indicate Yes, and report the date of the most recent assessment and specify if documentation of mental development neurocognitive testing was submitted to the CIBMTR. It is highly encouraged to attach this assessment / documentation.

If testing was not done or it is unknown, indicate No or Unknown, respectively and continue to the signature line.

For instructions on how to attach documents in FormsNet3SM, refer to the Training Guide.

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