Table 1.
Use of Sirolimus in Management of Neonatal Cardiac Rhabdomyoma
| First Author (Year), Country (Ref. #) | Case(s) | Treatment (Age, Dose, Trough Levels, Duration) | Monitoring | Prophylactic Co-Trimoxazole? | Results, Follow-Up |
|---|---|---|---|---|---|
| Breathnach et al. (2014), Ireland (4) | Term neonate (born 38 of 40 weeks), LVOT obstruction due to cardiac rhabdomyoma. | Treatment started day 10 of life, 0.5 mg oral daily (7 days), reduced to 0.4 mg oral daily due elevated trough level (26 ng/ml). Treated until 34 days of age (total 24 days). | Trough level, full blood count, electrolytes, renal and liver function, triglycerides. | Yes | Decrease in size of LVOT tumor by day 5 of treatment; “dramatic reduction” by day 24 of treatment. Treatment ceased at 34 days of age (day 24). Follow-up to age 8 months, stable LVOT gradient. |
| Lee et al. (2017), South Korea (3) | Premature neonate (born 28 of 40 weeks, birth weight 1.170 kg), LVOT obstruction due to cardiac rhabdomyoma. | Treatment started day 18 of life (CGA 30 + 4, weight 2.2 kg), 0.25 mg oral daily (14 days), reduced to 0.12 mg oral daily due to elevated trough level (42.1 ng/ml). Treated until 75 days of age (total 57 days). | Trough level, full blood count, electrolytes, renal and liver function, lipid profiling. | Yes | Decrease in size of LVOT tumor at day 15 of treatment, further reduction noted at days 22 and 43 of treatment. Treatment ceased at 75 days of age (day 57). Symptom-free at follow-up to 7 months of age. |
| Pendse and Deepika (2017), Australia (5) | Neonate with cardiac rhabdomyoma in intraventricular septum. | Not mentioned. | Not mentioned. | Not mentioned. | Cardiac arrest at day 29 of life, deceased. |
| Weiland et al. (2017), United States (6) | Two cases of neonatal cardiac rhabdomyoma. Case 1: Cardiac rhabdomyoma in apex of heart encroaching on left and right ventricle cavities. Case 2: LVOT obstruction due to cardiac rhabdomyoma. |
Case 1: Treatment started with 0.3 mg (0.1 mg/kg) daily. Trough level at 4 weeks post-commencement elevated 22.5 ng/ml. Case 2: Treatment started with 0.1 mg/kg twice daily, reduced to 0.05 mg/kg/dose at day 12 of treatment as trough level elevated (24.3 ng/dl), further reduced to 0.03 mg/kg/dose after 1 month of treatment as trough level elevated (12.1 ng/dl), resulting level 9.1 ng/dl. |
Trough level, other monitoring not specified. | Not mentioned. | Case 1: 74% reduction in tumor volume by day 11 of treatment, further reduction by 12% at 4 weeks. Interval size increase at follow-up 9 months after sirolimus cessation. Case 2: Decrease in size of tumor by day 12 of treatment (to approximately one-fourth of original size), LV free wall tumor decreased by >50%. |
CGA = corrected gestational age; LN = left ventricular; LVOT = left ventricular outflow tract.