Appeal type: Medical necessity
Decision: Overturned

Diagnosis: Cystinuria.
Treatment:  Thiola EC 300MG TAB, Pre-service.
The insurer denied the  Thiola EC 300MG TAB, Pre-service.
The denial is overturned. 

The patient is a male with documented cystinuria and cystine stones based on stone analysis and 24 hour urine studies.  There was initial question of a stone approximately six years ago, stone was identified on ultrasound (US) approximately four years ago and the patient has undergone ureteroscopy and laser lithotripsy for large stones on both left and right side approximately a year ago and again four months ago. The patient has been treated with aggressive hydration, dietary restriction and urocit potassium (K) ten milliequivalents (meq) three times daily (TID) with the most recent 24 hour urine study demonstrating elevated cystine at 732, potential of hydrogen (pH) of 7.322 and low sodium excretion. The patient's most recent ultrasound (US) demonstrated multiple small non-obstructing stones in the right kidney and at least two stones in the left. The patient was noted to have an elevated prothrombin time (PT) of 15.4 with normal international normalized ratio (INR) of 1.2 and received vitamin K at time of stone intervention. Request is for Thiola EC 300 milligrams (MG); 100 milligram tabs for treatment of his cytinuria and recurrent stones.

Yes, the proposed treatment with Thiola is medically necessary.
This male has cystinuria proven by stone analysis and elevated cystine in his urine of repetitive 24 hour urine studies. He has been placed on urocit potassium (K), high fluid intake, low salt and purine diet but remains with recurrent stone disease and persistent cystinuria despite improved urine potential of hydrogen (pH) (7.322) and low salt excretion (71) and has required multiple surgical interventions for obstructing stones. 

The requested treatment, Thiola EC, is Food and Drug Administration (FDA) approved.   Per the American Urologic Association (AUA) Guidelines, a first line agent for cystinuria refractory to hydration, urinary alkalinization and dietary modifications is Thiola. Thus, the requested use of Thiola would be considered standard of care for this patient and it would be considered medically necessary.

The patient was denied Thiola as the Plan noted that the patient has not tried penicillamine. The American Urologic Association (AUA) guidelines indicate penicillamine is associated with a higher incidence of side effects than Thiola.  Therefore, the requested Thiola is medically necessary.

The plan initially denied the use of Thiola based on the patient's history of elevated prothrombin time (PT) for which there is no documentation in the prescribing information that an elevated prothrombin time (PT) is a contraindication to the use of Thiola. The use of Thiola was also denied due to "not FDA approved"; however, Thiola EC was approved for the treatment of cystinuria in adult and pediatric patients greater than or equal to (>/=) 20 kilograms (Kg) unresponsive to urinary alkalinization, restrictive diet and hydration. The only listed contraindication to Thiola is patients with hypersensitivity to tiopronin or other components. The last denial noted "as no patient specific contraindication noted and no prior trial with penicillamine" the medication was denied and penicillamine was recommended. There is ample literature including the American Urological Association guidelines to support that Thiola is the preferred therapy due to its success rate and decreased incidence of adverse effects.  One article stated "Penicillamine is a well-known heavy metal chelator, classically used in the treatment of Wilson disease, rheumatoid arthritis, and cystinuria. From a dermatologic standpoint, penicillamine was found to be useful in the treatment of systemic sclerosis. The successful therapeutic uses of penicillamine have been hindered by its numerous adverse effects, both cutaneous and extra-cutaneous", Thus, Thiola is a therapy that is more tolerable, has less adverse effects and possible better efficacy than the plan recommended penicillamine.  Therefore, the requested Thiola is medically necessary.