Management and monitoring are essential elements of the treatment journey

Prior to infusion, ensure patients have baseline tests for liver function,a creatinine, complete blood count (including hemoglobin and platelet count), and troponin-I.1

After infusion, continue to monitor liver function, platelet count,b and troponin-I as described in the Prescribing Information. See the recommended schedule of assessments in the accompanying chart.1

Wk=week.

aLiver function assessment includes a clinical exam and laboratory testing of hepatic aminotransferases (aspartate aminotransferase [AST] and alanine aminotransferase [ALT]), total bilirubin, and prothrombin time. Monitor liver function for 3 months or longer until results are unremarkable (normal clinical exam, total bilirubin, and prothrombin results, and ALT and AST levels below 2 × ULN).

bMonitor platelet counts as well as signs and symptoms of TMA, such as hypertension, increased bruising, seizures, or decreased urine output.

cMonitor platelet counts and troponin-I levels for 3 months or longer until they return to baseline.

Table: Required tests to perform at baseline and after treatment with ZOLGENSMA including complete blood count, creatinine, platelet count, troponin-I, and liver function

=monitoring performed

aLiver function assessment includes a clinical exam and laboratory testing of hepatic aminotransferases (aspartate aminotransferase [AST] and alanine aminotransferase [ALT]), total bilirubin, and prothrombin time. Monitor liver function for 3 months or longer until results are unremarkable (normal clinical exam, total bilirubin, and prothrombin results, and ALT and AST levels below 2 × ULN).

bMonitor platelet counts as well as signs and symptoms of TMA, such as hypertension, increased bruising, seizures, or decreased urine output.

cMonitor platelet counts and troponin-I levels for 3 months or longer until they return to baseline.

Treat with systemic corticosteroids

To help manage a possible increase in liver aminotransferases, treat all patients with systemic corticosteroids before treatment and continue regimen after treatment1:

The recommended treatment regimen is detailed in the Prescribing Information and in the accompanying chart.

If oral corticosteroid therapy is not tolerated, consider intravenous corticosteroids as clinically indicated.

Day 1: 24 hours before infusion

Initiate 30-day, systemic corticosteroid regimen equivalent to oral prednisolone at 1 mg/kg/day

Day 2: Infusion Day

Infuse ZOLGENSMA and continue the corticosteroid regimen

Day 30 & Beyond

Do not stop systemic corticosteroids abruptly

If liver function findings are unremarkable (including AST & ALT <2 x ULN)*:

 

  • Taper the corticosteroid dose gradually over the next 28 days

If liver function abnormalities persist*:

 

  • Continue systemic corticosteroids until AST & ALT values are both below 2 x ULN and all other assessments return to normal range, and then taper the dose gradually over the next 28 days or longer if needed
  • Consult a pediatric gastroenterologist or hepatologist

*Monitor liver function for at least 3 months following ZOLGENSMA infusion and until results are unremarkablea

ALT=alanine aminotransferase; AST=aspartate aminotransferase; ULN=upper limit of normal.

aLiver function assessment includes a clinical exam and laboratory testing of hepatic aminotransferases (AST and ALT, total bilirubin, and prothrombin time). 

Ongoing care requires a multidisciplinary care team 

Even after disease-modifying treatment, patients with SMA may need respiratory, nutritional, and musculoskeletal support. The team will vary from patient to patient, but a coordinated team of specialists can help monitor the patient’s SMA.2-4

It is recommended that a neurological expert, such as a pediatric neurologist or a neuromuscular specialist, coordinate the care of the patient, as they will likely have the most experience with anticipating needs.3,4

While the specific needs and support levels of patients differ, part of the ongoing care can include an assessment of motor function, which may include regular sessions with a physical therapist and possible use of braces. Additionally, nutritionists can be involved to monitor weight and nutrient intake, paying careful attention to calcium and vitamin D for bone health.3

Multidisciplinary care team for treating patients with SMA includes neuromuscular specialists, respiratory, musculoskeletal, nutritional, and physical and occupational therapy

Patient and caregiver support

A resource for caregivers

Help caregivers understand what to expect after treatment and how to maintain proper SMA care. Fill out the Post-Treatment Plan and share it with your patient’s family prior to the patient being discharged.

Post-Treatment Plan

Patient Profiles

Explore the patient profiles and watch family stories

Patient Profiles

References: 1. ZOLGENSMA [prescribing information]. Bannockburn, IL: Novartis Gene Therapies, Inc; 2022. 2. Finkel RS, Mercuri E, Meyer OH, et al. Diagnosis and management of spinal muscular atrophy: part 2: pulmonary and acute care; medications, supplements and immunizations; other organ systems; and ethics. Neuromuscul Disord. 2018;28(3):197-207. 3. Mercuri E, Finkel RS, Muntoni F, et al. Diagnosis and management of spinal muscular atrophy: part 1: recommendations for diagnosis, rehabilitation, orthopedic and nutritional care. Neuromuscul Disord. 2018;28(2):103-115. 4. Wang CH, Finkel RS, Bertini ES, et al; Participants of the International Conference on SMA Standard of Care. Consensus statement for standard of care in spinal muscular atrophy. J Child Neurol. 2007;22(8):1027-1049.