- Email: [email protected]
Acupuncture for symptoms of Gaucher disease
Complementary Therapies in Medicine (2012) 20, 131—134
Available online at www.sciencedirect.com
journal homepage: www.elsevierhealth.com/journals/ctim
Acupuncture for symptoms of Gaucher disease Noah Samuels a,∗, Deborah Elstein b, Ehud Lebel b, Ari Zimran b, Menachem Oberbaum a a
Center for Integrative Complementary Medicine, Shaare Zedek Medical Center, Jerusalem, Israel Gaucher Clinic, Shaare Zedek Medical Center, Jerusalem, Israel Available online 9 January 2012
b
KEYWORDS Gaucher disease; Acupuncture; Pain; Fatigue; Quality of life
Summary Objective: The purpose of this study was to examine the effect of acupuncture on bone/joint pain, headache and fatigue, as well as quality of life in patients with Gaucher disease (GD), within the framework of an integrated treatment programme. Methods: Patients with GD suffering from any of the above symptoms were offered a series of 10—12 weekly acupuncture treatment sessions. Prior to initiation of treatment, participants were asked to score the severity of pain, as well as to complete the Functional Assessment of Chronic Illness Therapy-Fatigue measure (FACIT-F) and the Medical Outcomes Study (MOS) Short-Form (SF) questionnaire. These tools were evaluated again at the end of the treatment period. Results: A total of 12 patients were evaluated. While the only pain outcome reduced by acupuncture was knee pain, a significant improvement was observed with respect to nearly all FACIT-Fatigue measures, including the Physical Well Being (PWB) subscales and the SF-12 Physical Composite Score (PCS), though not for the Mental Composite Score (MCS). Patients reported satisfaction with the treatment process, and no significant side effects were reported. Conclusion: Acupuncture may play a beneficial role for patients with GD when used in conjunction with conventional therapy, reducing fatigue and improving physical function. The preliminary finding of this observational study should encourage further research. © 2011 Elsevier Ltd. All rights reserved.
Gaucher disease (GD) is a rare autosomal-recessive genetic disorder caused by an inborn deficiency of the enzyme glucocerebrosidase, with a predilection among Ashkenazi Jews for the non-neuropathic form (type 1) of the disease.1 A deficiency of the enzyme glucocerebrosidase in these patients leads to the accumulation of glucocerebroside in multiple
∗ Corresponding author at: Center for Integrative Complementary Medicine, Shaare Zedek Medical Center, P.O. Box 3235, Jerusalem 91031, Israel. Tel.: +972 2 6666395; fax: +972 2 6666975. E-mail address: [email protected] (N. Samuels).
organ systems. Although GD patients may be asymptomatic,2 many suffer from fatigue, attributed to bone marrow infiltration with anaemia, and bone/joint pain, due to skeletal infiltration. Skeletal pathologies such as ‘bone crises’ in children and osteonecrosis with pathological fractures in adults can lead to severe disability, and all major joints are at risk for avascular necrosis, lesions or infarcts, and pathological fractures, some with vertebral collapse.3 Current treatment of GD consists of enzyme replacement therapy (ERT) with recombinant imiglucerase.4 ERT results in increased haemoglobin and platelet counts, reduction in spleen and liver volumes, increased bone density and improvement
0965-2299/$ — see front matter © 2011 Elsevier Ltd. All rights reserved. doi:10.1016/j.ctim.2011.12.006
132 Table 1
N. Samuels et al. Demographic and disease-specific characteristics of patients.
Patient #
Gender (m/f)
Age (years)
Genotype
Disease severity12
Splenectomy
Avascular necrosis
ERT
1 2 3 4 5 6 7 8 9 10 11 12
f f m m m f f f m m m f
43 59 21 27 42 37 26 58 62 22 54 50
1226/84GG 1226/1226 1226/1448 1226/1448 1226/? 1226/84GG 1226/1448 1226/1226 1226/245 1226/84GG 1226/? 1226/1448
Severe Mild Mild Mild Mild Severe Mild Moderate Mild Moderate Mild Mild
Yes No No No No Yes Yes No Yes No No No
Yes No No No Yes Yes Yes Yes Yes No No No
Yes No Yes Yes Yes Yes Yes Yes Yes Yes No No
Disease severity: mild = 0—10; moderate = 11—20; severe = 21—30. ERT, enzyme-replacement therapy.
of other parameters within 2—5 years.5 Still, fatigue and bone/joint pain do not always respond to therapy, and can result in significant disability.6 Acupuncture is an ancient Chinese treatment during which thin needles (diameter < 0.30 mm) are inserted into designated points along the skin (acupoints). According to traditional Chinese medicine (TCM) stimulation of these points can alter the flow of the body’s energy, or ‘Qi’ (pronounced ‘chee’), throughout the body via organ-related channels, termed ‘meridians’. Clinical research has shown acupuncture to be beneficial for a number of chronic painrelated conditions such as backache7 and headache,8,9 as well as cancer-related fatigue.10,11 The purpose of this study was to examine the effect of acupuncture on bone/joint pain, headache and fatigue, as well as quality of life in GD patients, within the framework of an integrated treatment programme.
Methods Participants and outcome measures The Gaucher clinic at the Shaare Zedek Medical Center in Jerusalem, Israel, provides medical care for GD patients throughout Israel. In 2009, the clinic, in conjunction with the hospital’s Center for Integrative Complementary Medicine, set up a programme offering acupuncture treatments to Gaucher patients with bone/joint pain, headache or fatigue. Patients with other symptoms, as well as those unable to complete the acupuncture treatment programme for medical or other reasons, were not accepted. Treatments were
Table 2
offered free of charge and with no obligation, on a weekly basis, for a total of 10—12 treatments. In addition to the recruitment of patients from the study centre’s Gaucher clinic, the programme was advertised on the Israel Gaucher Association website and presented at the annual meeting of the association. At the beginning of each treatment session, a short interview and physical examination, based on the principles of TCM, were conducted. Acupuncture points were then chosen on an individualised basis, according to the ‘Stems and Branches’ approach of TCM. Single-use, sterilised acupuncture needles (0.16 mm × 30 mm; DongBang Acupuncture Inc., Korea) were used, with acupuncture treatments lasting from 30 to 40 min each time. Before beginning treatment, participants were asked to voluntarily score the severity of bone/joint pain and headache using a Likert-like numerical rating scale (range: 0—10 points), as well as completing the Functional Assessment of Chronic Illness Therapy-Fatigue measure (FACIT-F; www.facit.org) and the Medical Outcomes Study (MOS) Short-Form (SF) questionnaire (QualiMetric Incorporated, Lincoln, RI, US). Participants were asked to complete these same tools again at the end of the treatment period. In addition to the study tools mentioned above, participants were also asked to score (on a scale of 1—5) their satisfaction with the integrated treatment programme and outcome, as well as whether they would like to continue treatment, and whether they would recommend acupuncture to other patients with GD. Disease-related parameters, such as haemoglobin levels, genetic typing, history of splenectomy and avascular necrosis (AVN), and disease severity,12 were culled from patient records. The study
Quality of life (SF-12) scores at baseline and following acupuncture treatment.
Parameter (range)
Baseline mean ± SD (range)
After treatment mean ± SD (range)
P-value
Physical Composite Score Mental Composite Score
34.4 ± 11.89 (21.7—55.2) 45.3 ± 7.87 (34—59)
42.3 ± 10.27 (28.7—60.2) 48.5 ± 11.25 (27.3—62.7)
0.005 0.208
Acupuncture for symptoms of Gaucher disease Table 3
133
Fatigue (FACIT-F) scores at baseline and following acupuncture treatment.
Parameter (range)
Baseline mean ± SD (range)
After treatment mean ± SD (range)
P-value
Physical Well-Being (PWB; 0—28) Social Well-Being (SWB; 0—28) Emotional Well-Being (EWB; 0—24) Functional Well-Being (FWB; 0—28) Additional Concerns (AC; 0—52) Trial Outcome Index* (TOI; 0—108)
17.7 ± 6.14 (8—25) 21.8 ± 3.05 (16.3—28) 15.6 ± 4.98 (5—23) 18.2 ± 4.53 (9—23) 27.6 ± 8.30 (13—37) 63.2 ± 17.16 (37—88)
22.8 ± 3.22 (17—26) 20.0 ± 5.02 (8.4—26) 18.2 ± 3.41 (12—23) 20.3 ± 3.55 (15—26) 35.9 ± 6.16 (27—46) 80.6 ± 12.70 (59—100)
0.002 0.086 0.01 0.036 0.008 0.006
*
Trial Outcome Index (TOI) = PWB + EWB + SWB + FWB + A.
was approved by the Institutional Review Board (Helsinki Committee) of the research medical centre, with patients consenting to the use of the resulting data.
Statistical analysis
the benefit was not statistically significant (p = 0.086). Fig. 1 shows the effect of treatment on physical functioning as seen in both the SF-12 (PCS) and FACIT-F Physical Well-Being (PWB) subscales. Other than a slight discomfort with the odd needle insertion, no side effects of acupuncture were reported (Table 3).
Data were entered and managed using the Microsoft Excel 2003 Program, and later analysed using the SPSS Program, version 16 (SPSS, Inc., Chicago, IL, USA). To assess change in the quantitative outcome variables, particularly responses to the SF-12 and FACIT-F tools, a paired t-test was applied. The Wilcoxon signed-rank test was applied to ordinal variables (pain scores) as well as to quantitative variables in order to validate results from the paired t-test, this because of the small sample size. The association between changes in the SF-12 and FACIT-F scores and age was assessed by calculating the non-parametric Spearman correlation coefficient. The non-parametric Mann—Whitney test was used to test the association between SF-12 and FACIT-F scores with gender and ERT therapy. Finally, to test the association between the two questionnaires and severity of illness (3 sub-groups), the Kruskal—Wallis non-parametric analysis of variance (ANOVA) test was performed. All tests applied were two-tailed, and a value of p < 0.05 was considered statistically significant.
Results A total of 12 GD patients — six males and six females — participated in the programme, undergoing between 10 and 12 acupuncture treatments over the course of 10—21 weeks. All participants were of Ashkenazi (Jewish) origin, and the mean age was 41.75 years (SD: ±15.08). The demographic and disease-specific characteristics of the participants are presented in Table 1. Prior to treatment all but one patient (#4) reported musculoskeletal pain, with seven reporting hip pain (range: 2—9), of whom six had been diagnosed with AVN of the hip joint. Seven patients complained of knee pain (range: 2—8) and most had more than one painful joint. Four participants reported suffering from backache (range: 4—7). No significant improvement was found between the pre- and post-treatment haematological parameters, hip or hand pain, backache or headache following acupuncture treatment, although pain was reduced in those reporting knee pain (p = 0.028). A significant post-treatment benefit was noted for the SF-12 physical score (PCS; p < 0.0005), though not for the mental (MCS) score (p = 0.208). Nearly all FACIT-F subscales improved significantly following treatment (Table 2), though for the Social Well-Being (SWB) scale
Figure 1 The effect of acupuncture on physical outcome scores in Gaucher patients. (A) Medical Outcomes Study (MOS) Short-Form 12-question (SF-12), Physical Composite Score (PCS). (B) Functional Assessment of Chronic Illness Therapy — Fatigue (FACIT-F), Physical Well Being (PWB) subscale. For both tools, a low score is indicative of a more severely impaired level of function.
134 All 12 patients reported satisfaction (mean score of 4.75; SD: 0.452) with the treatment programme, although they were less satisfied with the treatment outcome (mean score of 3.41; SD: 0.651). Nine participants expressed a desire to continue treatment, with two more wishing to continue if not for the long distance they had to travel. Ten of the 12 patients would recommend acupuncture treatment to other Gaucher patients, with the remaining two ‘unsure’ regarding this question.
Discussion In the present study, acupuncture was found to be a safe and acceptable adjuvant therapy in patients with GD, with a significantly improved physical functioning, as measured by both the SF-12 PCS and FACIT-F PWB subscales. Pain, however, was reduced only in those patients suffering from knee pain. Patients were extremely satisfied with the addition of acupuncture to their current treatment regimen, and the overwhelming majority would recommend acupuncture to other patients with GD. There are a number of limitations to the present study. The findings are observational, and the study tools were initially intended for the evaluation of the feasibility and overall benefits of the integrated programme, and not for use in a clinical study. The size of the sample is small, with only 12 patients, which significantly reduces the statistical power of the findings as well. Nevertheless, the findings point towards a possible role of acupuncture for an indication in which there is, to date, little or no data. Patients with GD are often young, and should be enabled to be productive members of society. Larger, controlled clinical trials are needed to evaluate the effect of acupuncture on pain, fatigue and function, as well as other outcome parameters in these patients with GD. The preliminary observational findings of this study should encourage such research.
Conflict of interest The authors state that there are neither actual nor potential conflicts of interest, including and financial or personal relationships with other people or organisations. No funding was provided for this study.
N. Samuels et al.
Acknowledgments We are grateful to Daniel Levine, Lic. Ac., for his skill and empathy in administering the acupuncture treatments. We are also grateful to Tali Bdolah-Abram, M.Sc., for her assistance with the statistical analysis of the data and helpful comments.
References 1. Knudson AG, Kaplan WD. Genetics of the sphingolipidoses. In: Aronson SM, Volk BW, editors. Cereb sphingolipid. New York: Academic Press; 1962. p. 395. 2. Azuri J, Elstein D, Lahad A, Abrahamov A, Hadas-Halpern I, Zimran A, et al. Asymptomatic Gaucher disease implications for large-scale screening. Genet Test 1998;2:297—9. 3. Itzchaki M, Lebel E, Dweck A, Patlas M, Hadas-Halpern I, Zimran A, et al. Orthopedic considerations in Gaucher disease since the advent of enzyme replacement therapy. Acta Orthop Scand 2004;75:641—53. 4. Barton NW, Brady RO, Dambrosia JM, Di Bisceglie AM, Doppelt SH, Hill SC, et al. Replacement therapy for inherited enzyme deficiency — macrophage-targeted glucocerebrosidase for Gaucher’s disease. N Engl J Med 1991;324:1464—70. 5. Pastores GM, Weinreb NJ, Aerts H, Andria G, Cox TM, Giralt M, et al. Therapeutic goals in the treatment of Gaucher disease. Semin Hematol 2004;41:4—14. 6. Giraldo P, Solano V, Pérez-Calvo JI, Giralt M, Rubio-Félix D. Spanish group on Gaucher disease. Quality of life related to type 1 Gaucher disease: Spanish experience. Qual Life Res 2005;14:453—62. 7. Manheimer E, White A, Berman B, Forys K, Ernst E. Metaanalysis: acupuncture for low back pain. Ann Intern Med 2005;142:651—3. 8. Sun Y, Gan TJ. Acupuncture for the management of chronic headache: a systematic review. Anesth Analg 2008;107:2038—47. 9. Linde K, Allais G, Manheimer E, Vickers A, White AR. Acupuncture for tension-type headache. Cochrane Database Syst Rev 2009;1. CD007587. 10. Vickers AJ, Straus DJ, Fearon B, Cassileth BR. Acupuncture of post chemotherapy fatigue: a phase II study. J Clin Oncol 2004;22:1731—5. 11. Molassiotis A, Sylt P, Diggins H. The management of cancerrelated fatigue after chemotherapy with acupuncture and acupressure: a randomized controlled trial. Complement Ther Med 2007;15:228—37. 12. Zimran A, Sorge J, Gross E, Kubitz M, West C, Beutler E. Prediction of severity of Gaucher’s disease by identification of mutations at DNA levels. Lancet 1989;334:349—52.
Available online at www.sciencedirect.com
journal homepage: www.elsevierhealth.com/journals/ctim
Acupuncture for symptoms of Gaucher disease Noah Samuels a,∗, Deborah Elstein b, Ehud Lebel b, Ari Zimran b, Menachem Oberbaum a a
Center for Integrative Complementary Medicine, Shaare Zedek Medical Center, Jerusalem, Israel Gaucher Clinic, Shaare Zedek Medical Center, Jerusalem, Israel Available online 9 January 2012
b
KEYWORDS Gaucher disease; Acupuncture; Pain; Fatigue; Quality of life
Summary Objective: The purpose of this study was to examine the effect of acupuncture on bone/joint pain, headache and fatigue, as well as quality of life in patients with Gaucher disease (GD), within the framework of an integrated treatment programme. Methods: Patients with GD suffering from any of the above symptoms were offered a series of 10—12 weekly acupuncture treatment sessions. Prior to initiation of treatment, participants were asked to score the severity of pain, as well as to complete the Functional Assessment of Chronic Illness Therapy-Fatigue measure (FACIT-F) and the Medical Outcomes Study (MOS) Short-Form (SF) questionnaire. These tools were evaluated again at the end of the treatment period. Results: A total of 12 patients were evaluated. While the only pain outcome reduced by acupuncture was knee pain, a significant improvement was observed with respect to nearly all FACIT-Fatigue measures, including the Physical Well Being (PWB) subscales and the SF-12 Physical Composite Score (PCS), though not for the Mental Composite Score (MCS). Patients reported satisfaction with the treatment process, and no significant side effects were reported. Conclusion: Acupuncture may play a beneficial role for patients with GD when used in conjunction with conventional therapy, reducing fatigue and improving physical function. The preliminary finding of this observational study should encourage further research. © 2011 Elsevier Ltd. All rights reserved.
Gaucher disease (GD) is a rare autosomal-recessive genetic disorder caused by an inborn deficiency of the enzyme glucocerebrosidase, with a predilection among Ashkenazi Jews for the non-neuropathic form (type 1) of the disease.1 A deficiency of the enzyme glucocerebrosidase in these patients leads to the accumulation of glucocerebroside in multiple
∗ Corresponding author at: Center for Integrative Complementary Medicine, Shaare Zedek Medical Center, P.O. Box 3235, Jerusalem 91031, Israel. Tel.: +972 2 6666395; fax: +972 2 6666975. E-mail address: [email protected] (N. Samuels).
organ systems. Although GD patients may be asymptomatic,2 many suffer from fatigue, attributed to bone marrow infiltration with anaemia, and bone/joint pain, due to skeletal infiltration. Skeletal pathologies such as ‘bone crises’ in children and osteonecrosis with pathological fractures in adults can lead to severe disability, and all major joints are at risk for avascular necrosis, lesions or infarcts, and pathological fractures, some with vertebral collapse.3 Current treatment of GD consists of enzyme replacement therapy (ERT) with recombinant imiglucerase.4 ERT results in increased haemoglobin and platelet counts, reduction in spleen and liver volumes, increased bone density and improvement
0965-2299/$ — see front matter © 2011 Elsevier Ltd. All rights reserved. doi:10.1016/j.ctim.2011.12.006
132 Table 1
N. Samuels et al. Demographic and disease-specific characteristics of patients.
Patient #
Gender (m/f)
Age (years)
Genotype
Disease severity12
Splenectomy
Avascular necrosis
ERT
1 2 3 4 5 6 7 8 9 10 11 12
f f m m m f f f m m m f
43 59 21 27 42 37 26 58 62 22 54 50
1226/84GG 1226/1226 1226/1448 1226/1448 1226/? 1226/84GG 1226/1448 1226/1226 1226/245 1226/84GG 1226/? 1226/1448
Severe Mild Mild Mild Mild Severe Mild Moderate Mild Moderate Mild Mild
Yes No No No No Yes Yes No Yes No No No
Yes No No No Yes Yes Yes Yes Yes No No No
Yes No Yes Yes Yes Yes Yes Yes Yes Yes No No
Disease severity: mild = 0—10; moderate = 11—20; severe = 21—30. ERT, enzyme-replacement therapy.
of other parameters within 2—5 years.5 Still, fatigue and bone/joint pain do not always respond to therapy, and can result in significant disability.6 Acupuncture is an ancient Chinese treatment during which thin needles (diameter < 0.30 mm) are inserted into designated points along the skin (acupoints). According to traditional Chinese medicine (TCM) stimulation of these points can alter the flow of the body’s energy, or ‘Qi’ (pronounced ‘chee’), throughout the body via organ-related channels, termed ‘meridians’. Clinical research has shown acupuncture to be beneficial for a number of chronic painrelated conditions such as backache7 and headache,8,9 as well as cancer-related fatigue.10,11 The purpose of this study was to examine the effect of acupuncture on bone/joint pain, headache and fatigue, as well as quality of life in GD patients, within the framework of an integrated treatment programme.
Methods Participants and outcome measures The Gaucher clinic at the Shaare Zedek Medical Center in Jerusalem, Israel, provides medical care for GD patients throughout Israel. In 2009, the clinic, in conjunction with the hospital’s Center for Integrative Complementary Medicine, set up a programme offering acupuncture treatments to Gaucher patients with bone/joint pain, headache or fatigue. Patients with other symptoms, as well as those unable to complete the acupuncture treatment programme for medical or other reasons, were not accepted. Treatments were
Table 2
offered free of charge and with no obligation, on a weekly basis, for a total of 10—12 treatments. In addition to the recruitment of patients from the study centre’s Gaucher clinic, the programme was advertised on the Israel Gaucher Association website and presented at the annual meeting of the association. At the beginning of each treatment session, a short interview and physical examination, based on the principles of TCM, were conducted. Acupuncture points were then chosen on an individualised basis, according to the ‘Stems and Branches’ approach of TCM. Single-use, sterilised acupuncture needles (0.16 mm × 30 mm; DongBang Acupuncture Inc., Korea) were used, with acupuncture treatments lasting from 30 to 40 min each time. Before beginning treatment, participants were asked to voluntarily score the severity of bone/joint pain and headache using a Likert-like numerical rating scale (range: 0—10 points), as well as completing the Functional Assessment of Chronic Illness Therapy-Fatigue measure (FACIT-F; www.facit.org) and the Medical Outcomes Study (MOS) Short-Form (SF) questionnaire (QualiMetric Incorporated, Lincoln, RI, US). Participants were asked to complete these same tools again at the end of the treatment period. In addition to the study tools mentioned above, participants were also asked to score (on a scale of 1—5) their satisfaction with the integrated treatment programme and outcome, as well as whether they would like to continue treatment, and whether they would recommend acupuncture to other patients with GD. Disease-related parameters, such as haemoglobin levels, genetic typing, history of splenectomy and avascular necrosis (AVN), and disease severity,12 were culled from patient records. The study
Quality of life (SF-12) scores at baseline and following acupuncture treatment.
Parameter (range)
Baseline mean ± SD (range)
After treatment mean ± SD (range)
P-value
Physical Composite Score Mental Composite Score
34.4 ± 11.89 (21.7—55.2) 45.3 ± 7.87 (34—59)
42.3 ± 10.27 (28.7—60.2) 48.5 ± 11.25 (27.3—62.7)
0.005 0.208
Acupuncture for symptoms of Gaucher disease Table 3
133
Fatigue (FACIT-F) scores at baseline and following acupuncture treatment.
Parameter (range)
Baseline mean ± SD (range)
After treatment mean ± SD (range)
P-value
Physical Well-Being (PWB; 0—28) Social Well-Being (SWB; 0—28) Emotional Well-Being (EWB; 0—24) Functional Well-Being (FWB; 0—28) Additional Concerns (AC; 0—52) Trial Outcome Index* (TOI; 0—108)
17.7 ± 6.14 (8—25) 21.8 ± 3.05 (16.3—28) 15.6 ± 4.98 (5—23) 18.2 ± 4.53 (9—23) 27.6 ± 8.30 (13—37) 63.2 ± 17.16 (37—88)
22.8 ± 3.22 (17—26) 20.0 ± 5.02 (8.4—26) 18.2 ± 3.41 (12—23) 20.3 ± 3.55 (15—26) 35.9 ± 6.16 (27—46) 80.6 ± 12.70 (59—100)
0.002 0.086 0.01 0.036 0.008 0.006
*
Trial Outcome Index (TOI) = PWB + EWB + SWB + FWB + A.
was approved by the Institutional Review Board (Helsinki Committee) of the research medical centre, with patients consenting to the use of the resulting data.
Statistical analysis
the benefit was not statistically significant (p = 0.086). Fig. 1 shows the effect of treatment on physical functioning as seen in both the SF-12 (PCS) and FACIT-F Physical Well-Being (PWB) subscales. Other than a slight discomfort with the odd needle insertion, no side effects of acupuncture were reported (Table 3).
Data were entered and managed using the Microsoft Excel 2003 Program, and later analysed using the SPSS Program, version 16 (SPSS, Inc., Chicago, IL, USA). To assess change in the quantitative outcome variables, particularly responses to the SF-12 and FACIT-F tools, a paired t-test was applied. The Wilcoxon signed-rank test was applied to ordinal variables (pain scores) as well as to quantitative variables in order to validate results from the paired t-test, this because of the small sample size. The association between changes in the SF-12 and FACIT-F scores and age was assessed by calculating the non-parametric Spearman correlation coefficient. The non-parametric Mann—Whitney test was used to test the association between SF-12 and FACIT-F scores with gender and ERT therapy. Finally, to test the association between the two questionnaires and severity of illness (3 sub-groups), the Kruskal—Wallis non-parametric analysis of variance (ANOVA) test was performed. All tests applied were two-tailed, and a value of p < 0.05 was considered statistically significant.
Results A total of 12 GD patients — six males and six females — participated in the programme, undergoing between 10 and 12 acupuncture treatments over the course of 10—21 weeks. All participants were of Ashkenazi (Jewish) origin, and the mean age was 41.75 years (SD: ±15.08). The demographic and disease-specific characteristics of the participants are presented in Table 1. Prior to treatment all but one patient (#4) reported musculoskeletal pain, with seven reporting hip pain (range: 2—9), of whom six had been diagnosed with AVN of the hip joint. Seven patients complained of knee pain (range: 2—8) and most had more than one painful joint. Four participants reported suffering from backache (range: 4—7). No significant improvement was found between the pre- and post-treatment haematological parameters, hip or hand pain, backache or headache following acupuncture treatment, although pain was reduced in those reporting knee pain (p = 0.028). A significant post-treatment benefit was noted for the SF-12 physical score (PCS; p < 0.0005), though not for the mental (MCS) score (p = 0.208). Nearly all FACIT-F subscales improved significantly following treatment (Table 2), though for the Social Well-Being (SWB) scale
Figure 1 The effect of acupuncture on physical outcome scores in Gaucher patients. (A) Medical Outcomes Study (MOS) Short-Form 12-question (SF-12), Physical Composite Score (PCS). (B) Functional Assessment of Chronic Illness Therapy — Fatigue (FACIT-F), Physical Well Being (PWB) subscale. For both tools, a low score is indicative of a more severely impaired level of function.
134 All 12 patients reported satisfaction (mean score of 4.75; SD: 0.452) with the treatment programme, although they were less satisfied with the treatment outcome (mean score of 3.41; SD: 0.651). Nine participants expressed a desire to continue treatment, with two more wishing to continue if not for the long distance they had to travel. Ten of the 12 patients would recommend acupuncture treatment to other Gaucher patients, with the remaining two ‘unsure’ regarding this question.
Discussion In the present study, acupuncture was found to be a safe and acceptable adjuvant therapy in patients with GD, with a significantly improved physical functioning, as measured by both the SF-12 PCS and FACIT-F PWB subscales. Pain, however, was reduced only in those patients suffering from knee pain. Patients were extremely satisfied with the addition of acupuncture to their current treatment regimen, and the overwhelming majority would recommend acupuncture to other patients with GD. There are a number of limitations to the present study. The findings are observational, and the study tools were initially intended for the evaluation of the feasibility and overall benefits of the integrated programme, and not for use in a clinical study. The size of the sample is small, with only 12 patients, which significantly reduces the statistical power of the findings as well. Nevertheless, the findings point towards a possible role of acupuncture for an indication in which there is, to date, little or no data. Patients with GD are often young, and should be enabled to be productive members of society. Larger, controlled clinical trials are needed to evaluate the effect of acupuncture on pain, fatigue and function, as well as other outcome parameters in these patients with GD. The preliminary observational findings of this study should encourage such research.
Conflict of interest The authors state that there are neither actual nor potential conflicts of interest, including and financial or personal relationships with other people or organisations. No funding was provided for this study.
N. Samuels et al.
Acknowledgments We are grateful to Daniel Levine, Lic. Ac., for his skill and empathy in administering the acupuncture treatments. We are also grateful to Tali Bdolah-Abram, M.Sc., for her assistance with the statistical analysis of the data and helpful comments.
References 1. Knudson AG, Kaplan WD. Genetics of the sphingolipidoses. In: Aronson SM, Volk BW, editors. Cereb sphingolipid. New York: Academic Press; 1962. p. 395. 2. Azuri J, Elstein D, Lahad A, Abrahamov A, Hadas-Halpern I, Zimran A, et al. Asymptomatic Gaucher disease implications for large-scale screening. Genet Test 1998;2:297—9. 3. Itzchaki M, Lebel E, Dweck A, Patlas M, Hadas-Halpern I, Zimran A, et al. Orthopedic considerations in Gaucher disease since the advent of enzyme replacement therapy. Acta Orthop Scand 2004;75:641—53. 4. Barton NW, Brady RO, Dambrosia JM, Di Bisceglie AM, Doppelt SH, Hill SC, et al. Replacement therapy for inherited enzyme deficiency — macrophage-targeted glucocerebrosidase for Gaucher’s disease. N Engl J Med 1991;324:1464—70. 5. Pastores GM, Weinreb NJ, Aerts H, Andria G, Cox TM, Giralt M, et al. Therapeutic goals in the treatment of Gaucher disease. Semin Hematol 2004;41:4—14. 6. Giraldo P, Solano V, Pérez-Calvo JI, Giralt M, Rubio-Félix D. Spanish group on Gaucher disease. Quality of life related to type 1 Gaucher disease: Spanish experience. Qual Life Res 2005;14:453—62. 7. Manheimer E, White A, Berman B, Forys K, Ernst E. Metaanalysis: acupuncture for low back pain. Ann Intern Med 2005;142:651—3. 8. Sun Y, Gan TJ. Acupuncture for the management of chronic headache: a systematic review. Anesth Analg 2008;107:2038—47. 9. Linde K, Allais G, Manheimer E, Vickers A, White AR. Acupuncture for tension-type headache. Cochrane Database Syst Rev 2009;1. CD007587. 10. Vickers AJ, Straus DJ, Fearon B, Cassileth BR. Acupuncture of post chemotherapy fatigue: a phase II study. J Clin Oncol 2004;22:1731—5. 11. Molassiotis A, Sylt P, Diggins H. The management of cancerrelated fatigue after chemotherapy with acupuncture and acupressure: a randomized controlled trial. Complement Ther Med 2007;15:228—37. 12. Zimran A, Sorge J, Gross E, Kubitz M, West C, Beutler E. Prediction of severity of Gaucher’s disease by identification of mutations at DNA levels. Lancet 1989;334:349—52.