Questions 6 – 7: 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 neurologic function scale (NFS) score was obtained during the current reporting period. This information will be documented within a physician’s note.
If a total NFS score was obtained, select Yes, and report the date (YYYY-MM-DD) of assessment. If the total NFS score was obtained multiple times prior in the reporting period, 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 within the reporting period or it is not known if a score was obtained, indicate No, and continue with Did post-infusion seizures attributed to the underlying disease occur.
Question 8: 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 9 – 24: 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 a 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 25: Did post-infusion seizures attributed to the underlying disease occur since the date of the last report?
Indicate if the recipient had post-infusion seizures attributed to the underlying disease at any time during the current reporting period.
Unless otherwise noted by the physician, it can be assumed seizures were due to primary disease. Report No in the following scenarios and continue with Was cerebrospinal fluid (CSF) testing performed:
- The recipient did not have any seizures during the current reporting period
- The recipient experienced seizures during the current reporting period but they were not attributed to the underlying disease, as noted by the physician
If it is not known if the recipient had any seizures during the current reporting period or if the seizures were related to the underlying disease, select Unknown and continue with Was cerebrospinal fluid (CSF) testing performed.
Question 26: Were any of the seizures considered nonfebrile?
Specify if any of the seizures in the current reporting period were considered nonfebrile (spontaneous recurrent seizures unrelated to fever). If at least one seizure in the reporting period was nonfebrile, select Yes. If all seizures within the reporting period were febrile, or it is not known if all seizures were febrile, indicate No or Unknown, respectively.
Questions 27 – 29: Was cerebrospinal fluid (CSF) testing performed?
Indicate if cerebrospinal fluid (CSF) testing was completed within the current reporting period. CSF is collected via lumbar puncture or spinal tab. If CSF testing was not performed within the reporting period or it is not known, select No or Unknown, respectively and continue with Was magnetic resonance imaging (MRI) performed. The unknown option should be used sparingly and only when no judgment can be made to determine if this assessment was completed.
If CSF testing was performed select Yes and specify if the date (YYYY-MM-DD) of the most recent CSF testing is Known or Unknown. If Known, report the date of the most recent CSF assessment. If date is Unknown, continue with Specify known CSF result(s).
Questions 30 – 32: Specify known CSF result(s) (check all that apply)
Report the known CSF results, select all that apply.
- 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.
- 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 the rare case where neither the opening pressure or the total protein was known, leave Specify known CSF result(s) blank, override the validation error using the code “unknown,” and continue with Was magnetic resonance imaging (MRI) performed.
Questions 33 – 35: Was Magnetic Resonance Imaging (MRI) performed
Magnetic resonance imaging (MRI) is an imaging technique used to form pictures of the anatomy and the physiological processes of the body. MRIs are used to assess recipients with leukodystrophy.
If an MRI was performed during the current reporting period, select Yes and specify if the date (YYYY-MM-DD) of the most recent MRI is Known or Unknown. If Known, report the most recent MRI’s date. If the date is Unknown, continue with Specify MRI results.
If an MRI was not performed in the reporting period or it is not known, select No or Unknown, respectively and continue with Were nerve conduction velocities tested. The unknown option should be used sparingly only when no judgment can be made to determine if this assessment was completed.
Question 36: Specify MRI results
Specify the results of the most recent MRI performed in the reporting period. Indicate if the results were Normal or Abnormal as determined by the radiologist or physician.
Questions 37 – 38: 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. Report Yes or No if gadolinium contrast was used in the MRI assessment.
If gadolinium contrast was used, select Yes, and indicate if gadolinium enhancement was reported. 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.
Question 39: Loes composite score (ALD recipients only)
The Loes composite score is often used to assess disease / progression for recipients with ALD. The Loes composite score is a rating from 0-34, this signifies the severity of abnormalities detected in the brain after evaluation of MRI. This score should be listed within the MRI or within progress notes. Report the Loes composite score, if the score is unknown, check with a transplant physician to determine this value.
In the event where the transplant physician cannot determine the Loes composite score, leave the data field blank and override the FormsNet3SM error as “not documented.”
Question 40: Was documentation submitted to CIBMTR? (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 41 – 42: Were nerve conduction velocities tested
Indicate if nerve conduction velocities were tested at any time within the current reporting period. Nerve conduction velocity (NCV) testing measures how quickly an electrical impulse moves through the nerve and can identify nerve damage. This procedure is typically performed by a neurologist.
Indicate Yes, if NCV testing was performed prior within the reporting period and report the date (YYYY-MM-DD) of the most recent testing. If NCV testing was not done or it is unknown, report No or Unknown, respectively and continue with Was a neurocognitive test performed.
Questions 43 – 44: Specify Results
Indicate whether the results of the recipient’s NCV testing was Normal or Abnormal and 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 45 – 47: Was a neurocognitive test performed
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 within the reporting period. This information will be found within a progress note by the neuropsychologist.
If neurocognitive testing was performed, indicate Yes, and report the date (YYYY-MM-DD) of the most recent assessment. Specify if documentation of mental development neurocognitive testing was submitted to the CIBMTR. It is highly encouraged to attach this assessment / documentation. Additionally, complete the Neurocognitive Assessment (3503) Form.
If testing was not done or it is unknown, indicate No or Unknown, respectively and continue with Has there been a change in the recipient’s neurologic status.
For instructions on how to attach documents in FormsNet3SM, refer to the Training Guide.
Questions 48 – 50: Has there been a change in the recipient’s neurologic status (Report clinical status, not neuropsychological status).
Indicate if there has been a change in the recipient’s neurologic status since the previous reporting period. If Yes, specify if the current neurologic status has Improved or Worsened, when compared to the previous report. Additionally, specify if documentation of the physical or neurologic exam was submitted to the CIBMTR. It is highly encouraged to attach documentation of the exam from the neurologist or transplant physician.
For instructions on how to attach documents in FormsNet3SM, refer to the Training Guide.
If the recipient’s neurologic status is stable and / or unchanged since the previous reporting period, select Stable / unchanged.
If it is not known if the recipient’s neurologic status has changed (i.e., the physician’s progress notes are not clear if there has been a change in the neurologic status), select Unknown and continue with Specify global improvement.
Clinical Global Impression (CGI) (neurologic assessment)
Question 51: Specify global improvement (select one)
Report the recipient’s global improvement within the current reporting period. The Clinical Global Impression (CGI) is a 7-point scale, measuring the illness severity (CGIS), global improvement and / or change (CGIC) and therapeutic response. This information may not be documented within a routine follow-up progress note and will likely be found within a progress note by the neurologist or transplant physician.
If the recipient’s global improvement is not documented for the current reporting period, select Unknown.
Questions 52 – 53: Specify leukodystrophy-specific therapy given (check all that apply)
Select all leukodystrophy-specific treatment given during the current reporting period.
- N-acetyl-L-cysteine (NAC): (Acetylcysteine) is a supplement of cysteine (amino acid). Cysteine is an is an antioxidant agent that assists with respiratory conditions, fertility, and brain health.
- GTE:GTO oil (Lorenzo’s oil): Composed of erucic acid and oleic acids. Known to decrease / lower very long chain fatty acids (VLCA) levels.
- Other treatment: Includes those treatments not already listed above. If the recipient received treatments not listed, such as experimental clinical trials, select this option and specify the other treatment(s). Report the generic name of the agent, not the brand name.
If no therapy was given during the current reporting period, select None and go to the signature line.
Section Updates:
| Question Number | Date of Change | Add/Remove/Modify | Description | Reasoning (If applicable) |
|---|---|---|---|---|
| . | . | . | . |
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