Neuroprotective therapy using granulocyte colony-stimulating factor for patients with worsening symptoms of compression myelopathy, Part 1: a phase I and IIa clinical trial.

Abstract

OBJECTIVE:

Based on the neuroprotective effects of granulocyte colony-stimulating factor (G-CSF) on experimental spinal cord injury, we initiated a clinical trial that evaluated the safety and efficacy of neuroprotective therapy using G-CSF for patients with worsening symptoms of compression myelopathy.

METHODS:

We obtained informed consent from 15 patients, in whom the Japanese Orthopaedic Association (JOA) score for cervical myelopathy decreased two points or more during a recent 1-month period. G-CSF (5 or 10 μg/kg/day) was intravenously administered for five consecutive days. We evaluated motor and sensory functions of the patients and the presence of adverse events related to G-CSF therapy.

RESULTS:

G-CSF administration suppressed the progression of myelopathy in all 15 patients. Neurological improvements in motor and sensory functions were obtained in all patients after the administration, although the degree of improvement differed among the patients. Nine patients in the 10-μg group (n=10) underwent surgical treatment at 1 month or later after G-CSF administration. In the 10-μg group, the mean JOA recovery rates 1 and 6 months after administration were 49.9±15.1 and 59.1±16.3%, respectively. On the day following the start of G-CSF therapy, the white blood cell count increased to more than 22,700 cells/mm3. It varied from 12,000 to 50,000 and returned to preadministration levels 3 days after completing G-CSF treatment. No serious adverse events occurred during or after treatment.

CONCLUSION:

The results indicate that G-CSF administration at 10 μg/kg/day is safe for patients with worsening symptoms of compression myelopathy and may be effective for their neurological improvement.

• Authors: Tsuyoshi Sakuma, Masashi Yamazaki, Akihiko Okawa, Hiroshi Takahashi, Kei Kato, Mitsuhiro Hashimoto, Koichi Hayashi, Takeo Furuya, Takayuki Fujiyoshi, Junko Kawabe, Chikato Mannoji, Ryo Kadota, Masayuki Hashimoto, Kazuhisa Takahashi, Masao Koda
• Journal: European spine journal : official publication of the European Spine Society, the European Spinal Deformity Society, and the European Section of the Cervical Spine Research Society (Eur Spine J) Vol. 21 Issue 3 Pg. 482-9 (Mar 2012) ISSN: 1432-0932 [Electronic] Germany
• PMID: 21935680 (Publication Type: Case Reports, Clinical Trial, Phase I, Clinical Trial, Phase II, Journal Article, Research Support, Non-U.S. Gov't)

Chemical References

• Neuroprotective Agents
• Granulocyte Colony-Stimulating Factor

Topics

• Adult
• Aged
• Disease Progression
• Female
• Granulocyte Colony-Stimulating Factor (administration & dosage)
• Humans
• Injections, Intravenous (methods)
• Male
• Middle Aged
• Neuroprotective Agents (administration & dosage)
• Paresthesia (drug therapy, etiology, physiopathology)
• Quadriplegia (drug therapy, etiology, physiopathology)
• Recovery of Function (drug effects, physiology)
• Spinal Cord (drug effects, physiology)
• Spinal Cord Compression (drug therapy, etiology, physiopathology)
• Spondylosis (complications, physiopathology)
• Treatment Outcome