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At least 487 records · Page 27Linked to original sources
PubMed2004

Bipolar disorder. III: Harm avoidance a risk factor for suicide attempts.

OBJECTIVES: The aim of the study was to examine whether personality, i.e. temperament and character influence suicide attempts in bipolar patients. METHODS: Bipolar patients were recruited from lithium dispensaries. Temperament and character inventory (TCI) was administered to 100 euthymic bipolar patients and 100 controls. RESULTS: Age of onset was significantly lower in patients with suicide attempts in the total bipolar group (I and II) and bipolar I patients compared with patients without suicide attempts. Bipolar (I and II) and bipolar I patients with suicide attempts were significantly higher in harm avoidance (HA) and reward dependence compared with patients without suicide attempts. Patients (I and II) with suicide attempts had significantly more anticipatory worry, fatigability and asthenia than patients without suicide attempts. Bipolar I patients with suicide attempts had significantly more fatigability and asthenia and were more dependent than patients without suicide attempts. HA was lowest in patients with no suicide attempts and no family history of suicide, higher in patients with family history of suicide or patients with suicide attempts, and significantly highest in patients with suicide attempts and family history of suicide. Patients with suicide attempts and family history of suicide had more anticipatory worry, fatigability and asthenia. Bipolar disorder was significantly correlated to HA and suicide attempts to HA and PS. Family history of suicide and gender were significantly correlated to suicide attempts. CONCLUSIONS: Age of onset, HA, PS, gender and family history of suicide had a moderate to very strong effect on suicide attempts in bipolar patients.

Antipsychotic Agents↗
PubMedDate not supplied

Comparison between the GIRBAS Scale and the Acoustic and Aerodynamic Measures Provided by EVA for the Assessment of Dysphonia following Unilateral Vocal Fold Paralysis.

The aim of this study is to establish relevant objective parameters for evaluating dysphonia following unilateral vocal fold paralysis. To do so, the study compares objective and perceptual voice measures. The objective measures were obtained using a voice analysis software (Evaluation Vocale Assistée), whereas the perceptual measures were established with the GIRBAS Scale (grade, instability, roughness, breathiness, asthenia, and strain). All measurements were performed on 40 voice samples: 28 dysphonic subjects with unilateral laryngeal paralysis, and 12 control subjects. The intra- and inter-judge agreements were fairly good, at least for control subjects. The six GIRBAS measures obtained from the pathological voices were higher than those from the control voices (p < 0.001) and the correlation between both groups was good. Grade, breathiness and asthenia correlated with the objective parameters that express the aperiodicity of the phonatory signal (p < 0.01), namely, the coefficient of variability of the fundamental frequency, the coefficient of variability of intensity, and jitter. Our findings suggest that the perceptual reality of laryngeal paralysis-induced dysphonia depends more on grade, breathiness and asthenia than it does on roughness or instability.

Female↗
PubMed1998

Phase I trial of docetaxel administered by weekly infusion in patients with advanced refractory cancer.

PURPOSE: Docetaxel is a highly active antineoplastic agent; however, grade IV leukopenia occurs in the large majority of patients treated with a dose of 100 mg/m2 every 3 weeks. Recent experience with weekly paclitaxel has demonstrated a bone marrow-sparing effect when a weekly administration schedule is used. We investigated a weekly schedule of docetaxel in an attempt to alter the toxicity profile and improve the therapeutic index. PATIENTS AND METHODS: Thirty-eight patients with advanced, refractory malignancy entered this phase I trial between October 1996 and June 1997. Docetaxel was administered weekly for 6 consecutive weeks, followed by 2 weeks without treatment. Sequential cohorts of patients were treated at the following dose levels: 20, 25, 30, 36, 43, and 52 mg/m2. Patients were reevaluated after one course (8 weeks); patients with objective response or stable disease continued treatment for a maximum of four courses or until disease progression. RESULTS: Thirty-five patients completed at least one course of therapy. Myelosuppression was not a dose-limiting toxicity (DLT) at any of the doses tested. Only five episodes of grade III leukopenia occurred (14% of patients, 2% of doses), and no grade IV leukopenia was produced. No grade III or IV thrombocytopenia or anemia was observed. Grade III fatigue and asthenia were observed in all three patients treated at 52 mg/m2/wk and in two of 10 at 43 mg/m2/wk. Other grade III toxicity included acral erythema (n = 1), neuropathy (n = 1), peripheral edema (n = 1), and diarrhea (n = 1). The DLTs of this docetaxel schedule are fatigue and asthenia. Although the maximum-tolerated dose by definition of this study was 43 mg/m2/wk, we selected 36 mg/m2/wk for ongoing phase II studies. CONCLUSION: The toxicity profile of docetaxel is markedly altered when the drug is administered by a weekly schedule. Myelosuppression is mild and uncommon. Fatigue and asthenia are the DLTs; other nonhematologic toxicities, which included peripheral edema and neuropathy, are uncommon, and the arthralgia/myalgia syndrome was not observed. Weekly administration of docetaxel may provide a better tolerated, efficacious use of this drug; further investigation of weekly docetaxel as a single agent and in combination regimens is warranted.

Adult↗
PubMed2000

Phase I-II study of paclitaxel, cisplatin, and gemcitabine in advanced transitional-cell carcinoma of the urothelium. Spanish Oncology Genitourinary Group.

PURPOSE: To determine the maximum-tolerated dose and the antitumor activity of a combination of paclitaxel, cisplatin, and gemcitabine in advanced transitional-cell carcinoma (TCC) of the urothelium. PATIENTS AND METHODS: Patients with measurable, previously untreated, locally advanced or metastatic TCC and with Eastern Cooperative Oncology Group performance status < or = 2 and creatinine clearance > or = 55 mL/min were eligible. Cisplatin was given on day 1 at a fixed dose of 70 mg/m(2). Paclitaxel and gemcitabine were given on days 1 and 8 at increasing dose levels. Cycles were repeated every 21 days to a maximum of six cycles. RESULTS: Sixty-one patients were registered. In phase I, 15 patients were entered at four different dose levels. Dose-limiting toxicity consisted of early onset (after the first cycle) grade 2 asthenia (two of six patients) and grade 3 asthenia (one of six patients) at dose level 4. A paclitaxel dose of 80 mg/m(2) and gemcitabine 1,000 mg/m(2) was recommended for phase II, and 46 additional patients were entered at this level for a total of 49 patients. Main nonhematologic toxicity was grade 2 asthenia in 18 patients, with early onset in five patients, and grade 3 in four patients. Grade 3/4 neutropenia and thrombocytopenia occurred in 27 (55%) and 11 (22%) patients, respectively. Overall, febrile neutropenia was seen in 11 patients, and one toxic death occurred because of neutropenic sepsis. The combination was active at all dose levels. In total, 58 of 61 eligible patients were assessable for response; 16 complete responses (27.6%) and 29 partial responses (50%) were observed for an overall response rate of 77.6% (95% confidence interval, 60% to 98%). The median survival time (MST) available for the phase I part of the study is 24.0 months. MST has not been reached for the whole group with the current follow-up. CONCLUSION: This combination of paclitaxel, cisplatin, and gemcitabine is feasible and highly active in patients with advanced TCC of the urothelium. Further evaluation of this regimen in patients with TCC is warranted.

Adult↗
PubMed2000

The mechanism of adverse events associated with terazosin: an analysis of the Veterans Affairs cooperative study.

PURPOSE: We determined the mechanism of adverse events associated with alpha1-blockers for treating benign prostatic hyperplasia (BPH). MATERIALS AND METHODS: We randomized 1,229 men with clinical BPH at 31 Veterans Affairs medical centers into equal treatment groups, including those who received placebo, terazosin, finasteride, and combined terazosin and finasteride therapy, respectively. Adverse events were captured at all study visits during our 1-year study. Our current review of adverse events is limited to patients randomized to the placebo and terazosin groups. We compared the incidence of orthostatic blood pressure change, postural symptoms and orthostatic hypotension in men who were normotensive and hypertensive at baseline, respectively. We also determined the association of changes in systolic blood pressure with the incidence of treatment related adverse events. RESULTS: The treatment related rates of dizziness, asthenia, postural hypotension and syncope were 19%, 6%, 6% and 1%, respectively. Of these adverse events only postural hypotension was associated with orthostatic blood pressure changes. The incidence of asthenia, dizziness and postural hypotension was not significantly greater in patients with a systolic blood pressure decrease of 5 or greater and less than 5 mm. Hg, respectively. CONCLUSIONS: Dizziness and asthenia are not associated with changes in blood pressure, suggesting that these treatment related adverse events associated with alpha1-blockers are not related to vascular events. Designing a subtype selective alpha1 antagonist that has less effect on blood pressure may not result in marked improvement in tolerability over commercially available alpha1-blockers.

Adrenergic alpha-Antagonists↗
PubMed2002

Therapeutic approaches for chronic gastralgia based on differentiation of symptoms and signs.

OBJECTIVE: To report our clinical experience with the treatment of chronic gastralgia on the basis of the differentiation of symptoms and signs. METHOD: A total of 160 cases of chronic gastralgia we treated in recent years were reviewed. RESULTS: Among the 160 cases, 120 falls in the category characterized by the predominant clinical symptoms such as depression of liver-energy and spleen-asthenia. For the other 40 cases, their clinical symptoms are of deficiency of stomach-yin, and retention of damp-heat in the interior or accumulation of blood stasis constituted the major clinical presentations, but more or less featured by depression of liver-energy and spleen-asthenia. Through analysis by differentiation of the symptoms and signs, we were convinced that the major symptoms of chronic gastralgia and some accompanying minor symptoms were all the representation and extension of the underlying cause, disorder of stomach-energy. The treatment was therefore targeted at dispersing the depression of liver-energy and invigorating the spleen, smoothing and regulating the stomach-energy. The treatment adopts primary prescription and good effects were achieved. CONCLUSION: The pathogenesis of chronic gastralgia predominantly lies in the depression of liver-energy and spleen-asthenia, together with disordered stomach-energy. The treatment should be implemented to disperse the depression of liver-energy and invigorate the spleen, with attention to smoothing and regulating the stomach-energy throughout the whole treatment course.

Chronic Disease↗
PubMed1992

[Reduction of genotoxic damage in oral mucosa with myasthenia gravis by traditional Chinese medicine].

Micronucleated exfoliated cell (MEC) of oral mucosa of 40 patients with myasthenia gravis (MG) and 54 normal controls were observed by means of micronucleus test. The frequency of MEC of two groups were 9.56/1000 and 2.55/1000 respectively, and their difference was remarkably significant (P < 0.001). 22 cases were treated by TCM Qiang Ji Jian Li Capsule. The frequency of MEC after treatment fell from 14.38/1000 to 6.00/1000. The difference was significant (P < 0.002). The frequency of Spleen-asthenia group higher than that of non-Spleen-asthenia (P < 0.05). The results revealed that. The patients with MG had genotoxic damage. (2) The constitution with the Spleen-asthenia and the genotoxic damage was related. (3) Qiang Ji Jian Li Capsule of invigorating the Spleen and benefiting Qi could reduce the genotoxic damage.

Adolescent↗
PubMed2006

Hormonal treatment for prostate cancer in Italy. Preliminary data from a survey of the QUABIOS Group.

OBJECTIVE: An observational study was planned by the QUABIOS group, to survey the hormonal modalities administered to prostate cancer patients in Italy within a time window of 12 months. We report here a summary of treatment schedules and related adverse effects, as recorded at the first visit. MATERIAL AND METHODS: Patients with diagnosis of prostate cancer and under hormonal therapy (LHRH-a and/or antiandrogen, previous orchidectomy) were eligible for this study. Adverse events were reported (graduated according to NCI-CTC v2.0, when pertaining) only in patients having received at least three months of hormonal treatment. RESULTS: 560 patients were enrolled from February to December, 2004 by 33 urological centers in Italy. A moderate to severe impairment of sexual function subsequent to the hormonal treatment was observed in 18.3% of patients treated with antiandrogen monotherapy, in 48.1% of patients treated with LHRH-a, and in 59.9% of patients treated with the combined approach. 24.7% patients referred a gastrointestinal toxicity (nausea/vomiting and diarrhea), without clear differences between treatment modalities. An asthenia subsequent to the hormonal treatment was moderate to severe in 3.5% of patients treated with antiandrogen monotherapy, as compared to 14.5% for LHRH-a and 12.9% for the combined approach, respectively. A gynecomastia / breast pain was present in 27.8% of antiandrogen monotherapy patients, as compared to 13.9% for LHRH-a and 13.8% for the combined approach, respectively. As compared to cyproterone acetate, bicalutamide had more sexual function impairment, more gastrointestinal toxicity, (slightly) more asthenia and more gynecomastia / breast pain. CONCLUSIONS: In our series antiandrogens were obviously associated with less sexual function impairment and less asthenia than LHRH-a containing schedules; on the other hand gynecomastia mainly affected patients receiving antiandrogen monotherapy. Within antiandrogens, cyproterone acetate generally appeared to be better tolerated than bicalutamide, with less severe impairment of sexual function, less gastrointestinal toxicity and negligible gynecomastia.

Aged↗
PubMed2006

[Cytomegalovirus infection with hepatic involvement in immunocompetent adults].

We retrospectively evaluated 73 immunocompetent adult patients assisted at our Infectious Diseases Clinic between March 1999 and March 2004 who presented fever and asthenia, mild to moderate increase of transaminases and serological findings compatible with recent cytomegalovirus infection. We excluded patients with a history of transfusions, drug abuse, immunodeficiencies, preexistent hepatic impairment or serological findings compatible with acute hepatitis A, B and C (HAV, HBV, HCV) and Epstein-Barr virus (EBV). The laboratory diagnosis of recent cytomegalovirus infection was made by especific IgM detection (ELISA) or a significant increase of specific IgG. The most frequent symptoms were fever (85%) and asthenia (83%), followed by cephalea (25%), splenomegaly (20%), adenomegalies (22%), pharyngitis (25%), myalgias (25%) and hepatomegaly (19%). All the patients showed moderate increase of transaminases and lymphomonocytosis (73/73). In average, ALT was increased by 6 fold and AST by 3.5 fold. The clinical characteristics that differentiate CMV infection from Epstein-Barr infection are the lesser frequency of adenomegalies and pharyngitis in the former. The differential diagnosis of CMV infection with hepatic involvement from acute hepatitis A and B, is based on the absence of jaundice, the lower elevation of transaminases, the intense lymphomonocytosis and the presence of specific IgM against CMV that are characteristic of CMV infection. In conclusion, in previously healthy young adults with fever, intense asthenia, lymphomonocytosis and moderate increase in transaminases levels, cytomegalovirus infection should be investigated.

Adolescent↗
PubMed2006

[Possible mechanisms of chronic fatigue syndrome in multiple sclerosis].

One of frequent presentations of multiple sclerosis (MS) is chronic fatigue that may be determined as a subjective decrease of the physic and/or psychic energy level. Fatigue can be divided into asthenia (fatigue in resting state), pathological fatigability (exhaustion during physical loading) and fatigue concomitant with other symptoms (MS exacerbation). There are central as well as peripheral mechanisms of fatigue formation. Frequent is a combination of fatigue and affective disorders in MS, in particular depression, as well as sleep disturbances (insomnia, restless legs syndrome) that may indicate the common origin of their mechanisms, i.e. reduction of serotoninergic and noradrenergic systems activity. Endocrinal and autoimmune components are considered as important in fatigue syndrome formation, the latter exerting more influence on asthenia than on pathological fatigability. Further investigation into pathogenetic mechanisms of asthenia (fatigue in resting state), pathological fatigability (fatigue in active state) and specification of their differential diagnostic features allow not only to understand the essence of this syndrome but to choose an adequate individualized therapy.

Fatigue Syndrome, Chronic↗
PubMed1993

[Asthenic-autonomic disorders in the decompensation phase of residual organic brain conditions].

The paper provides characteristics of asthenic and vegetative-vascular defects in decompensation of residual organic cerebral insufficiency. The defects were examined vegetologically with the help of photoplethysmography, thermovisiography, the test of the vegetative, psychological, exercise performance. Cutaneous-galvanic reflex, attention, memory, asthenia severity were also evaluated. The data obtained by the authors are important for making differential diagnosis of cerebrogenic with psychogenic asthenia in neurosis which allows differential therapy of asthenia symptoms depending on the etiological factor.

Adolescent↗
PubMedDate not supplied

[Comparative double-blind study of bromazepam versus prazepam in non-psychotic anxiety].

The efficacy of bromazepam and prazepam for the different components of anxiety: inhibition, asthenia and somatisation is evaluated in a multi-centric, comparative and randomised study, conducted as double blind and in parallel groups in 159 adult patients showing a manifest anxiety according to the F.D.A. criteria. After a 7 day wash-out period, the patients receive either bromazepam in a 12 mg/d dose or prazepam in a 40 mg/d dose, over 4 weeks (D0-D28), then in a decreasing dose from D28 to D43; follow-up is carried out using the anxious inhibition scale W.P.2, auto-questionnaire A.D.A., the Hamilton anxiety scale and the Tyrer questionnaire (benzodiazepine withdrawal symptoms questionnaire). Patients are evaluated seven times during the study: at day 7 for inclusion, day 0 for randomisation, then day 7 and day 14 for following visits, at day 28 for efficacy and tolerance evaluation, and at day 50 for utilisation and withdrawal evaluation. The major efficacy criteria are the evolution of inhibition, asthenia and somatisation as compounds of anxiety respectively evaluated by W.P.2 scale, asthenic partial score of autoquestionnaire A.D.A. and somatic partial score of Hamilton anxiety scale. The analysis of results don't show any significant difference between the two groups on the evolution of the components asthenia and inhibition. However the evolution of the somatic component clearly makes a significant difference in favour of bromazepam. There is also a significant difference in terms of global anxiolytic action efficacy, in favour of bromazepam.(ABSTRACT TRUNCATED AT 250 WORDS)

Adolescent↗
PubMedDate not supplied

Long-term prognosis of 69 patients with dermatomyositis or polymyositis.

OBJECTIVES: To assess the long-term prognosis of dermatomyositis and pol myositis. METHODS: 69 patients with dermatomyositis or polymyositis were selected according to the diagnostic criteria of Bohan and Peter and were followed up for a minimum of 6.3 years (for surviving patients) (mean 11.6 years). Clinical and biological features, and pulmonary and muscle parameters were considered as prognostic factors for death. Functional disability was assessed using a 4-stage grading system. RESULTS: 30 deaths (43.5%) occurred mainly due to cardiovascular (8), pulmonary (8), carcinomatous (5) and iatrogenic complications (5). Survival rates were 82.6% at 1 year, 73.9% at 2.66, 7% at 5 and 55.4% at 9. Significant prognostic factors for death (Cox model with time-dependent covariates) were old age (p < 0.0001), dysphonia (p < 0.001), pulmonary interstitial fibrosis (p < 0.02), absence of dysphagia (p < 0.02) and asthenia-anorexia (p < 0.05). Dermatomyositis and polymyositis subgroups had slightly different significant prognostic factors for death: old age, cancer, pulmonary interstitial fibrosis and asthenia-anorexia for dermatomyositis; old age, failure to improve muscle strength in response to treatment after one month, and the absence of myalgia as presenting symptom for polymyositis. At the end of the follow-up, 33/39 surviving patients (84.6%) had no or insignificant muscular disability, whereas 3 children were bedridden due to generalized calcinosis. CONCLUSIONS: High mortality occurred in the first year, and the survival rate decreased continually up to 9 years. The main prognostic factor for death is old age, but dermatomyositis and polymyositis must be considered separately. General features (pulmonary fibrosis, cancer, asthenia-anorexia) are involved in dermatomyositis, whereas muscular symptoms are the most significant in polymyositis. The long-term functional prognosis was fairly good, except for generalized calcinosis, which tended to occur in childhood dermatomyositis.

Adolescent↗
PubMed1998

[POEMS syndrome with primary adrenocortical insufficiency].

BACKGROUND: POEMS syndrome is a multisystem disorder. It usually presents as severe polyneuropathy and monoclonal gammapathy associated with endocrinopathies, organomegaly, skin hyperpigmentation. CASE REPORT: A patient with POEMS syndrome developed primary adrenocortical deficiency revelated by asthenia and hyperpigmentation. Hydrocortisone replacement therapy produced a rapidly beneficial effect on asthenia and hyperpigmentation. DISCUSSION: Although asthenia and skin pigmentation are common in POEMS syndrome, adrenocortical deficiency is seldom reported. This endocrinopathy might be underestimated in POEMS patients. We suggest systematic screening with a rapid ACTH test in all POEMS patients in order to detect underlying adrenal deficiency.

Adrenal Cortex Function Tests↗
PubMed1999

Ifosfamide and vinorelbine in metastatic breast cancer in patients with prior anthracycline therapy.

OBJECTIVE: A prospective trial to evaluate the efficacy and toxicity of ifosfamide (IFX) and vinorelbine (VNB) in patients with prior anthracycline therapy for metastatic breast cancer (MBC) was conducted. At the same time, the scheduling of VNB in order to minimize toxicity of the combination was also evaluated. PATIENTS AND METHODS: Twenty-three patients with MBC who had already received initial chemotherapy with doxorubicin-containing regimens either as adjuvant or as first-line treatment in MBC were entered into the study. IFX 3 g/m(2) and mesna 3 g/m(2) were given in divided doses over 2 days. In 4 patients, VNB was given 25 g/m(2) on days 1 and 3 in 3-h infusion. In 8 patients, VNB was given on days 1 and 8 and in 5 patients VNB was given on days 1 and 15. Thirteen patients had received doxorubicin in adjuvant setting, while 10 patients received doxorubicin as first-line treatment in metastatic disease. Dominant disease sites were soft tissues in 7 patients, visceral in 12 patients, and bone in 4 patients. The median age was 47 years. RESULTS: Overall objective response was seen in 12/23 patients (52.2%). Four patients achieved complete remission (CR), 8 patients achieved partial remission (PR). The median duration of response was 9 months in responding patients, and the median overall survival duration was 15 months. The major dose-limiting toxicities were neutropenia grade III and IV in 8/17 patients and asthenia grade III and IV in 4 patients. CONCLUSION: IFX and VNB is an active combination. Neutropenia and asthenia were most significant when VNB was given on days 1 and 3. In the best-tolerated regimen, VNB was given on days 1 and 8.

Adult↗
PubMed2005

Psychobiological personality dimensions in two environmental-illness patient groups.

The aim of the present study was to investigate the psychobiological personality dimensions in two subgroups of patients with environmental illness (EI). Fifty-nine patients, 34 women and 25 men (aged 32-69 years), were referred for symptoms allegedly caused by abnormal sensitivity to either dental fillings (DF; n=26) or electromagnetic fields (EMF; n=33). For the evaluation of personality, the Swedish 238-item version of the Temperament and Character Inventory (TCI) was used. Compared with a control group, the EMF group scored higher on the temperament dimension Persistence. The DF group scored higher on the TCI subscales Harm Avoidance (fatigability and asthenia) and Self-Directedness (self-acceptance). Women scored higher than men did on the Novelty Seeking and Reward Dependence (RD) dimensions in the DF group and on RD in the control group, indicating an inherited gender difference. No differences were found between men and women in the EMF group. Our results indicate that the high level of persistence found in the EMF group and the high level of fatigability and asthenia in combination with high self-acceptance found in the DF group represent vulnerable personalities. No significant differences were found between the two patient groups, indicating that these groups are quite similar regarding personality. This vulnerability can be expressed as various mental and somatic symptoms, which can be interpreted as EI symptoms by the affected individual.

Adult↗
PubMed1999

[Muscle, fatigue, sports and infection].

PURPOSE: Skeletal muscle can be considered as motors which convert chemical energy into mechanical energy. We can evaluate the intracellular pH and energy state of phosphate-containing metabolites in skeletal muscle of patients complaining fatigue or asthenia, using phosphorus MRS. MAIN POINTS: Acute infectious disease and extreme endurance exercise may induce a loss of oxidative capacity of muscle tissue. Muscle fatigue is not due only to an insufficient supply of ATP to the energy consuming mechanisms. Phosphorus MRS show a muscle production of toxic metabolites such as lactates, protons and ammonia. These metabolic features induced excessive intracellular acidosis of skeletal muscle and systemic hyperammonia, responsible of fatigue and asthenia. PERSPECTIVES: Reversal of the excessive acidosis and improvement of the capacity for oxidative ATP synthesis might help to relieve the symptoms of exhaustion/fatigue in these patients.

Animals↗
PubMed2006

[Clinical study of the French cohort of Gaucher disease patients].

A census is currently being carried out of the French cohort of Gaucher disease patients. This article describes its preliminary results, obtained by analysing the records of 101 patients for whom clinical and laboratory data were accessible while they were receiving enzyme therapy. At the time of diagnosis, all patients presented with splenomegaly, 70% had asthenia and one in three was already affected by major bone damage. After 1 year of enzyme therapy, splenomegaly had diminished by half and the different scores (asthenia, bone pain and abdominal pain, etc.) had markedly improved, as had the biochemical markers. As for the 6 patients affected by type 3 Gaucher disease and treated with enzyme therapy after the onset of neurological signs, a stabilisation or even some improvement of the disease was observed. In-depth study of the French cohort should make it possible to formulate consensus recommendations for the future, based on well-established data.

1-Deoxynojirimycin↗