Preoperative Acetazolamide Challenge in Pediatric Chiari I Malformation

In patients who present with headaches and Chiari 1 malformation without cranial nerve or brainstem dysfunction or syrinx formation, the decision to decompress can be difficult to make. We present a case series examining the use of acetazolamide as a diagnostic aid to determine candidacy for decompression.

A single pediatric neurosurgeon's (M.M.H.) experience from 2003 to 2018 was retrospectively reviewed. All cases evaluated in the clinic for CPT code of Chiari 1 malformation were analyzed. Inclusion criteria were patients with diagnosis of Chiari 1 malformation and headache-predominant symptoms who underwent an acetazolamide challenge test. Exclusion criteria included age over 18, syrinx, or significant clinical evidence of brainstem compression or cranial nerve dysfunction. Data were recorded with respect to response to acetazolamide. Surgical outcomes were assessed using the Chicago Chiari Outcome Scale (CCOS) pain category and the improve/same/worse (IWS) scale. Overall satisfaction from surgery was also recorded.

Forty-five patients met inclusion criteria and underwent acetazolamide challenge. Thirty-three (73.3%) patients showed improvement of their symptoms with acetazolamide challenge (responders). Of the 33 responders, 25 (75.8%) underwent Chiari decompression. The remaining 8 (24.2%) responders experienced persistent improvement of their symptoms after the acetazolamide trial and did not require intervention. Twelve (26.7%) patients did not improve with acetazolamide (nonresponders) of which 11 (91.7% of nonreponders) never required intervention. One nonresponder eventually underwent Chiari decompression due to progressively worsening and debilitating headaches. All twenty-five (100%) responders improved after surgery and 24 (96%) were satisfied with the outcome of surgery. The average responder CCOS pain score was 3.52 out of 4. The nonresponder who did not improve with acetazolamide had persistent severe headaches after decompression. The CCOS pain score for this nonresponder was 2 out of 4.

Pediatric headache-predominant Chiari 1 malformation presents a difficult diagnostic dilemma. The authors describe a trial of acetazolamide as a preoperative diagnostic tool to aid the decision to decompress. Further studies will need to be performed to determine the effectiveness of preoperative acetazolamide in identifying which patients may benefit from surgical decompression.