Table 7.

Experimental Targeted Treatment of Manifestations in Individuals with Bachmann-Bupp Syndrome

Targeted TreatmentDosageConsideration
Difluoromethylornithine (DFMO) 1
  • Escalating 500 mg/m2/dose orally BID for 3 mos to 750 mg/m2/dose orally for 3 mos
  • 1000 mg/m2/dose orally BID for maintenance
  • This dosing strategy was used for 3 patients w/BABS under FDA-approved Single Patient IND based on previous data from pediatric dosing for neuroblastoma treatment.
  • Future treatment strategies for DFMO may be influenced by ongoing safety & efficacy data.

BID = twice a day; FDA = Food and Drug Administration; IND = investigational new drug

1.

Also called eflornithine

From: Bachmann-Bupp Syndrome

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