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Peripheral neuropathy in Krabbe disease: effect of hematopoietic stem cell transplantation
Zaeem A Siddiqi1, Donald B Sanders, Janice M Massey
1Division of Neurology, University of Alberta Hospital, Edmonton, AB, Canada. zsiddiqi@ualberta.ca
Insights
Hematopoietic stem cell transplantation (HSCT) can improve peripheral nerve conduction in Krabbe disease (KD) patients, suggesting nerve remyelination. Early intervention shows greater benefit, though effects may be temporary.
Area of Science:
- Neurology
- Pediatric Neurology
- Stem Cell Transplantation
Background:
- Krabbe disease (KD) is a severe neurological disorder.
- Early hematopoietic stem cell transplantation (HSCT) may slow KD progression.
- Over 90% of KD patients exhibit significant peripheral nerve conduction abnormalities.
Purpose of the Study:
- To evaluate the impact of HSCT on peripheral nerve conduction in KD patients.
- To assess the potential for remyelination following HSCT in Krabbe disease.
Main Methods:
- Serial nerve conduction studies (NCS) were performed on 12 KD patients post-HSCT.
- Follow-up averaged 18 months, with a range of 6 months to 3 years.
- Pre-transplant NCS were available for 10 of 12 patients, all showing abnormalities.
Main Results:
- HSCT led to recordable sural sensory responses (SNR) in 3 patients and F-waves in 1 patient.
- Normal SNR was achieved in 7 of 12 patients post-HSCT.
- Improvements were observed in distal motor nerve latency, motor nerve conduction velocity (CV), and F-wave latencies (FWL), with earlier transplants yielding better results. Some abnormalities worsened over time, indicating potential temporary benefits.
Conclusions:
- Serial NCS are valuable for monitoring peripheral neuropathy in KD.
- HSCT demonstrates potential for improving peripheral nerve conduction abnormalities in KD, suggesting remyelination.
- The benefits of HSCT for nerve conduction in KD may be temporary, necessitating ongoing monitoring.
Background:
Hematopoietic stem cell transplantation (HSCT) may slow the progression of Krabbe disease (KD) if performed early in the disease. The authors' studies indicate that more than 90% of patients with KD have severe abnormalities in peripheral nerve conduction.
Objective:
To assess the effect of HSCT on nerve conduction in patients with KD.
Methods:
The authors performed serial nerve conduction studies (NCS) in 12 patients with KD after HSCT. The average follow-up was 18 months (6 months to 3 years) after HSCT. Pretransplant NCS were not available in two patients; all others (10 of 12) had significant pretransplant abnormalities.
Results:
After HSCT, previously absent F-waves (1 patient) and sural sensory responses (SNR) (3 patients) were found recordable. All patients continued to have recordable SNR after HSCT, and these became normal in 7 of 12 patients. Distal motor nerve latency became normal in 6 of 17 and motor nerve conduction velocity (CV) in 2 of 17 nerves; F-wave latencies (FWL) improved in 6 of 17 nerves, but did not become normal in any. There was greater improvement in nerve conduction abnormalities if the transplant was performed earlier in life. After an initial improvement, there was subsequent worsening of motor latencies (2 of 12), motor CV (2 of 12), FWL (3 of 12), and SSR (1 of 12), indicating that benefit from HSCT may be temporary.
Conclusions:
Serial nerve conduction studies are useful in following the course of peripheral neuropathy in Krabbe disease. Hematopoietic stem cell transplantation is followed by improvement in peripheral nerve conduction abnormalities in these patients, suggesting remyelination of the nerves.
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