Effective treatment of painful bone crises in type I gaucher's disease with high dose prednisolone.

Abstract

In type I Gaucher's disease, episodes of severe disabling bone pain, the so called bone crises, may be resistant to all analgesics, including narcotics. The demonstration of subperiosteal oedema on magnetic resonance imaging (MI) led to an attempt to use steroids to relieve the oedema and thereby the pain. On eight occasions, five patients with documented bone crises received conventional dose steroids (20 mglm2lday) with considerable shortening of the attacks. On six occasions five further patients received high dose methylprednisolone (30 mg/kg intravenously or 1 gIm2 orally daily for two days), which was followed by oral prednisone for three to five days on the last four occasions. In this latter group, pain reliefwas evident within several hours. Three treatments were given on an ambulatory basis. The M1Il scan of one of these patients showed no subperiosteal fluid collection five days after high dose steroids had been started, and on subsequent x ray examination, there was no periosteal elevation. This treatment should be considered in cases of Gaucher's disease with bone crises.(Arch Dis Child 1996;75:218-222) (total 18 episodes). Patients who presented with acute onset of severe bone pain underwent clinical examination, routine blood studies, and blood cultures. An immediate technetium-99m methylene diphosphonate (MDP) bone scan was performed. Bone crisis was diagnosed by the demonstration of decreased MDP uptake in the painful area.4 If this study was not helpful, magnetic resonance imaging (MRI) was performed, and a high intramedullary and subperiosteal signal on Ti and T2 weighted sequences was considered diagnostic of bone crisis.`Since osteomyelitis can show a similar clinical picture, it is imperative to rule out this condition by the bone scan, which would show increased uptake in this situation.

Pediatric non-neuronopathic Gaucher disease: presentation, diagnosis and assessment. Consensus statements

“…This suggests that the pain is caused by the periosteal elevation secondary to the fluid collection. The finding that in some cases, high-dose, shortterm use of corticosteroids causes absorption of the fluid, prevention of the periosteal elevation and pain relief in hours would support this view [13].…”

Paediatric non-neuronopathic Gaucher disease: recommendations for treatment and monitoring

“…Infarctions, bone crises, fractures and osteonecrosis may be accompanied by severe and debilitating pain. Analgesic options include acetaminophen and non-steroidal anti-inflammatory drugs and, although narcotic analgesics have been cited as options for severe and limited events such as bone crises, they are not effective in all patients [16]. An alternative treatment is high-dose oral prednisolone, which has been reported to reduce periosteal oedema and provide relief of severe pain within hours -even pain refractive to morphineduring bone crises [16].…”

“…Analgesic options include acetaminophen and non-steroidal anti-inflammatory drugs and, although narcotic analgesics have been cited as options for severe and limited events such as bone crises, they are not effective in all patients [16]. An alternative treatment is high-dose oral prednisolone, which has been reported to reduce periosteal oedema and provide relief of severe pain within hours -even pain refractive to morphineduring bone crises [16]. Concerns that high-dose steroids may cause bone disease (especially avascular necrosis) have been unsubstantiated [14,15], possibly because of the acute nature of the treatment.…”

Lysosomal storage disease overview

“…The extent of bone marrow infiltration, osteosclerosis, and osteonecrosis can be assessed by MRI of the spine and femur, and bone mineral density by (dual-energy X-ray absorption) DEXA scan. Acute bone crises can be refractory to narcotics, but high dose steroids may relieve pain (53). Therapeutic goals and management guidelines are available for Gaucher disease type I (44,45,54,55).…”