Klin Padiatr 2010; 222 - DGPI_PO_20
DOI: 10.1055/s-0030-1261436

Micafungin – Overview of Efficacy in Pediatric Patients

A Groll 1, NL Seibel 2, TJ Walsh 3, L Arnold 4, A Arrieta 5
  • 1Westf. Wilhelms-Univ.- Kinderklinik, Münster
  • 2Children's National Medical Center, Washington, DC, USA
  • 3National Cancer Institute, Bethesda, MD, USA
  • 4Astellas Pharma, Deerfield, ILL, USA
  • 5Children's Hospital of Orange County, Orange, CA, USA

Background and aim: Invasive fungal infections are a major source of morbidity and mortality in immunocompromized children; however, information on new antifungal therapies in children is limited. We evaluated the efficacy and pharmacokinetics (PK) of micafungin, a novel antifungal agent, by reviewing pediatric data from micafungin clinical trials. Methods: A retrospective review of pediatric data from clinical trials was performed. Results: A total 296 children received micafungin (MICA) for invasive candidiasis (IC), refractory IC, refractory invasive aspergillosis (IA), prophylaxis in hematopoietic stem cell transplantation (HSCT) patients, or to assess PK. Most patients age <1 year were premature (38/66), whereas most children age >1 year were HSCT recipients or malignancy patients (181/230). Median maximum daily dose for patients <1, 1 to 4, 5 to 8, 9 to 12 and 13 to 15 years were 2.0, 1.5, 1.5, 1.9 and 1.5mg/kg, respectively. Treatment success rates are shown in the table. MICA showed linear PK, with a higher clearance in neonates than in older children and adults.

Conclusions: MICA is an efficacious agent for the treatment and prophylaxis of pediatric invasive fungal infections.

Therapeutic Category

Treatment

N

Treatment Response, n/N (%)
by Age Group (years)

0 to 2

3 to 7

8 to 12

13 to <16

Overall

Phase III

Invasive candidiasis or candidemia

Micafungin

52

24/30

(80.0)

5/7

(71.4)

3/8

(37.5)

4/7

(57.1)

36/52 (69.2)

Liposomal Amphotericin B

54

31/40

(77.5)

6/9

(66.7)

1/3

(33.3)

2/2

(100)

40/54 (74.1)

Prophylaxis of fungal infections

Micafungin

39

6/7

(85.7)

10/14 (71.4)

7/12 (58.3)

4/6

(66.7)

27/39 (69.2)

Fluconazole

45

6/13 (46.2)

4/7

(57.1)

10/19 (52.6)

4/6

(66.7)

24/45 (53.3)

Phase II

Invasive aspergillosis

Micafungin*

58

2/5

(40.0)

7/16 (43.8)

5/18 (27.8)

12/19 (63.2)

26/58 (44.8)

Invasive candidiasis or candidemia

Micafungin

53

19/28 (67.9)

7/11 (63.6)

8/9

(88.9)

4/5

(80.0)

38/53 (71.7)

Phase I

PK, safety, and tolerability

Micafungin

69

4/5

(80.0)

23/30 (76.7)

13/22 (59.1)

9/12 (75.0)

49/69 (71.0)

*Either alone or in combination.