- Treatment of Spinal Muscular Atrophy (SMA)
- Available as 12 mg/5 mL (2.4 mg/mL) vial - clear colorless solution
- Store refrigerated at 2 C to 8 C. Do not freeze. Protect from light. Keep in original packaging up to time of administration
- If in original packaging, protected from light and at room temperature (less than 30 C) -stable 14 days
- If removed from packaging and at room temperature (less than 25 C) - stable 30 hours
- Vial should be taken out of refrigerator and allowed to warm to room temperature without using external heat sources, prior to administration. The solution must be clear and colorless. Do not use if visible particles observed or discolored.
- Once in syringe - stable 6 hours.
- Discard unused solution left in the vial
- DO NOT MIX with other drugs or solutions
(For approved routes of administration by nursing personnel, refer to Policy for the Administration of Intravenous Medications.)
| SC | NO |
| IM | NO |
| IV direct | NO |
| IV intermittent infusion | NO |
| IV continuous infusion | NO |
| Intrathecal |
YES - must remove 5 mL cerebrospinal fluid prior to administration
|
(For neonatal dosages, refer to Neonatal IV Drug Manual.)
Pediatric:
- 12 mg (5 mL) / administration
- Initiation (4 loading doses): 12 mg as an intrathecal bolus on Days 0, 14, 28 and 58
- If a loading dose is missed or delayed - administer as soon as possible with at least 14 days between doses 1 to 3 and 30 days between the third and fourth bolus dose - Maintenance: 12 mg as an intrathecal bolus every 4 months
- If a maintenance dose is missed or delayed it should be given as soon as possible and continue as previously scheduled
Adult:
- Limited data in patients greater than 18 years. Studies only in newborn to 19 years of age.
Dosage Adjustments:
Renal impairment:
- has NOT been studied in renal impairment so no dosage recommendation for adjustment exists.
- highly concentrated in the kidneys proximal tubules and excreted by the kidney so should not be used in renal impairment.
Hepatic impairment:
- has NOT been studied in hepatic impairment
- NOT metabolized via cytochrome P450 so dosage adjustment is unlikely to be required
Potential hazards of Intrathecal administration:
- Post lumbar puncture syndrome (CNS leak, headache)
- Infection
Adverse reactions:
- Thrombocytopenia
- Vomiting
- Constipation
- Headache
- Back pain
- Pyrexia
- Upper respiratory tract infections
- Upper respiratory tract congestion
- Atelectasis and hypoxia
- Dermatitis
- Metabolized via nucleases NOT cytochrome P450 pathway
Monitoring:
- Platelet count: At baseline and prior to each dose
- Prothrombin time and activated prothrombin time: At baseline and prior to each dose
- Urine protein analysis: baseline and monthly