Severe progressive scoliosis in an adult female possibly secondary thoracic surgery in childhood treated with scoliosis specific Schroth physiotherapy: Case presentation

Background

Scoliosis is a complex three-dimensional (3D) spinal deformity. Acquired scoliosis in early childhood may progress into adulthood and pose an increased risk of health problems and reduction in quality of life. In Canada, patients with scoliosis are not referred for physiotherapeutic scoliosis-specific exercises (PSSE) despite the fact that Schroth physiotherapy, a scoliosis-specific 3D posture training and exercise program, can be effective in reducing pain and improving scoliosis curves, vital capacity, and overall quality of life in scoliosis patients. This case presentation shows that indeed adult curve progression can be stopped and even reversed with scoliosis specific Schroth physiotherapy (SSSPT) in an adult patient with scoliosis.

Methods

This is a retrospective case presentation involving a 23-year-old female scoliosis patient who began an outpatient Schroth physiotherapy exercise program and was initially monitored monthly and then annually for improvement in measurements of angle of trunk rotation (ATR) and chest expansion and improvement in vital capacity measured with incentive spirometry. Photos were taken to document body image periodically throughout Schroth physiotherapy treatment. Additionally, the patient completed SRS-22 quality of life questionnaires every 2 years to evaluate daily function, pain, self-imagine, mental health, and scoliosis management satisfaction.

Results

Within one month of beginning SSSPT, the patient reported no more back pain and within 2 months, reported improved breathing. The patient also benefitted from improved chest expansion, reduced scoliosis curve angles (measured in Cobb degrees), increased vital capacity, decreased ATR, and higher SRS-22 scores. She became more active and resumed all athletic activity within 8 months of beginning Schroth physiotherapy.

Conclusions

Adult scoliosis patients are not routinely referred for PSSE in Canada, even though Schroth physiotherapy, a form of PSSE, is shown to be effective in this case presentation. The patient in this case presentation was successfully treated with Schroth physiotherapy. Long-term comprehensive Schroth physiotherapy, to help correct and maintain proper posture in all aspects of daily living, should be part of scoliosis management for adult scoliosis patients in Canada to stop and reverse curve progression and to improve overall quality of life.

     

Inhibition of p38-MAPK signaling pathway attenuates breast cancer induced bone pain and disease progression in a murine model of cancer-induced bone pain

Background

Mechanisms driving cancer-induced bone pain are poorly understood. A central factor implicated to be a key player in the process of tumorigenesis, osteoclastogenesis and nociception is p38 MAPK. We determined the role of p38 MAPK in a mouse model of breast cancer induced bone pain in which mixed osteolytic and osteoblastic remodeling occurs.

Results

In cancer-treated mice, acute as well as chronic inhibition of p38 MAPK with SB203580 blocked flinching and guarding behaviors in a dose-dependent manner whereas no effect on thresholds to tactile stimuli was observed. Radiographic analyses of bones demonstrated that chronic inhibition of p38 MAPK reduced bone loss and incidence of spontaneous fracture in cancer-treated mice. Histological analysis of bones collected from mice treated with the p38 MAPK inhibitor showed complete absence of osteoblastic growth in the intramedullary space as well as significantly reduced tumor burden.

Conclusions

Blockade of non-evoked pain behaviors but not hypersensitivity suggests differences in the underlying mechanisms of specific components of the pain syndrome and a possibility to individualize aspects of pain management. While it is not known whether the role of p38 MAPK signaling can be expanded to other cancers, the data suggest a need for understanding molecular mechanisms and cellular events that initiate and maintain cancer-induced bone pain for effective management for both ongoing pain as well as breakthrough pain.     

Factors associated with pain level in non-cardiac chest pain patients with comorbid panic disorder

Background

Panic disorder (PD) is highly prevalent in patients with non-cardiac chest pain (NCCP). This study aims to explore the role of psychological factors (PD intensity, anxiety sensitivity, heart-related fear, attention and avoidance) common to NCCP and PD in predicting chest pain levels in patients with both conditions.

Methods

This association was investigated in emergency department patients with NCCP and PD receiving either evidence-based treatment of PD or treatment as usual. Patients were assessed at baseline and 14 weeks later for post-treatment.

Results

Only heart-focused fear and attention for cardiac sensations independently explained a significant portion of the variance in baseline pain (n?=?66). At 3 months follow-up (n?=?53), changes in heart-related fear was the only factor independently associated with changes in chest pain intensity. Even in patients with PD, fear specific to cardiac sensations seems to play a central role in determining NCCP intensity.

Conclusion

These results suggest that the efficacy of intervention for patients with PD and comorbid NCCP could be improved by targeting heart-related fear and attention.

Trial registration

NCT00736346

     

Gelatin microspheres containing calcitonin gene-related peptide or substance P repair bone defects in osteoporotic rabbits

Objectives

To investigate the therapeutic effect of gelatin microspheres containing different concentrations of calcitonin gene-related peptide (CGRP) or substance P on repairing bone defects in a rabbit osteoporosis model.

Results

Gelatin microspheres containing different concentrations of CGRP or substance P promoted osteogenesis after 3 months in a rabbit osteoporotic bone defective model. From micro-computed tomography imaging results, 10 nM CGRP was optimal for increasing the trabecular number and decreasing the trabecular bone separation degree; similar effects were observed with the microspheres containing 1 µM substance P. Histological analysis showed that the gelatin microspheres containing CGRP or substance P, regardless of the concentration, effectively promoted osteogenesis, and the highest effect was achieved in the groups containing 1 µM CGRP or 1 µM substance P.

Conclusions

Gelatin microspheres containing CGRP or substance P effectively promoted osteogenesis in a rabbit osteoporotic bone defect model dose-dependently, though their effects in repairing human alveolar ridge defects still need further investigation.

     

<Emphasis Type="Italic">CER1</Emphasis>gene variations associated with bone mineral density,bone markers,and early menopause in postmenopausal women

Background

Osteoporosis has a multifactorial pathogenesis characterized by a combination of low bone mass and increased fragility. In our study, we focused on the effects of polymorphisms in CER1 and DKK1 genes, recently reported as important susceptibility genes for osteoporosis, on bone mineral density (BMD) and bone markers in osteoporotic women. Our objective was to evaluate the effect of CER1 and DKK1 variations in 607 postmenopausal women. The entire DKK1 gene sequence and five selected CER1 SNPs were amplified and resequenced to assess whether there is a correlation between these genes and BMD, early menopause, and bone turnover markers in osteoporotic patients.

Results

Osteoporotic women seem to suffer menopause 2 years earlier than the control group. The entire DKK1 gene sequence analysis revealed six variations. There was no correlation between the six DKK1 variations and osteoporosis, in contrast to the five common CER1 variations that were significantly associated with BMD. Additionally, osteoporotic patients with rs3747532 and rs7022304 CER1 variations had significantly higher serum levels of parathyroid hormone and calcitonin and lower serum levels of osteocalcin and IGF-1.

Conclusions

No significant association between the studied DKK1 variations and osteoporosis was found, while CER1 variations seem to play a significant role in the determination of osteoporosis and a potential predictive role, combined with bone markers, in postmenopausal osteoporotic women.

     

Evidence based guidelines for complex regional pain syndrome type 1

Background  

Treatment of complex regional pain syndrome type I (CRPS-I) is subject to discussion. The purpose of this study was to develop multidisciplinary guidelines for treatment of CRPS-I.     

An unusual cause of chest pain:case report

Background

Sarcomas form a heterogenous group of relatively uncommon malignant tumours which are derived from connective tissue components. In total they comprise approximately 1% of all new cancers diagnosed per year in the United Kingdom (UK). As subset of this, the 'Unclassified' Sarcoma forms approximately 4% of the total [1]. They often present with as relatively slow growing, asymptomatic masses and as such may often be misdiagnosed as in this case.

Case presentation

A 52 year old man presented to his general practitioner (GP) with left sided chest pain. A strong family history of ischaemic heart disease prompted hospital referral and further investigations which all proved negative for coronary artery disease. Following weight loss and ongoing chest pain, he represented to his GP with a hard mass arising from the left pectoralis major muscle at the site of the previous pain. Surgical excision followed by later compartectomy revealed an unclassified low grade Sarcoma with lymphoma like features.

Conclusion

In this case, chest pain masquerading as ischaemia, may have been caused by peri-neural infiltration or compression of adjacent muscle bulk by tumour, with eventual surgical resection providing a good long term prognosis.

     

Microbicides Development Programme: design of a phase III trial to measure the efficacy of the vaginal microbicide PRO 2000/5 for HIV prevention

Background

Non-pharmacological, non-surgical interventions are recommended as the first line of treatment for osteoarthritis (OA) of the hip and knee. There is evidence that exercise therapy is effective for reducing pain and improving function in patients with knee OA, some evidence that exercise therapy is effective for hip OA, and early indications that manual therapy may be efficacious for hip and knee OA. There is little evidence as to which approach is more effective, if benefits endure, or if providing these therapies is cost-effective for the management of this disorder. The MOA Trial (Management of OsteoArthritis) aims to test the effectiveness of two physiotherapy interventions for improving disability and pain in adults with hip or knee OA in New Zealand. Specifically, our primary objectives are to investigate whether: 1. Exercise therapy versus no exercise therapy improves disability at 12 months; 2. Manual physiotherapy versus no manual therapy improves disability at 12 months; 3. Providing physiotherapy programmes in addition to usual care is more cost-effective than usual care alone in the management of osteoarthritis at 24 months.

Methods

This is a 2 × 2 factorial randomised controlled trial. We plan to recruit 224 participants with hip or knee OA. Eligible participants will be randomly allocated to receive either: (a) a supervised multi-modal exercise therapy programme; (b) an individualised manual therapy programme; (c) both exercise therapy and manual therapy; or, (d) no trial physiotherapy. All participants will continue to receive usual medical care. The outcome assessors, orthopaedic surgeons, general medical practitioners, and statistician will be blind to group allocation until the statistical analysis is completed. The trial is funded by Health Research Council of New Zealand Project Grants (Project numbers 07/199, 07/200).

Discussion

The MOA Trial will be the first to investigate the effectiveness and cost-effectiveness of providing physiotherapy programmes of this kind, for the management of pain and disability in adults with hip or knee OA.

Trial registration

Australian New Zealand Clinical Trials Registry ref: ACTRN12608000130369.     

Younger and older chronic somatoform pain patients in psycho-diagnostics,physician-patient relationship and treatment outcome

Introduction

Patients with chronic pain are found with highly variable clinical presentation and differing physical complaints. They are seen as a heterogenic group. Based on clinical observations, elderly patients seem to differ from younger patients with chronic pain. We examined whether there were systematic differences between young and old pain patients.

Methods

As part of a routine evaluation of university hospital care, a newly developed psychosomatic treatment model for chronic somatoform pain disorders was examined. The basis for treatment efficacy was a target-oriented, specific somatic and psychological intervention that included a stable physician-patient relationship. Particular attention was paid to differences in treatment outcome with regard to changes in both physical and psychopathological symptom levels. We hypothesised that younger pain patients had higher psychological burden and benefitted more from our treatment than older pain patients.

Results

Overall, 179 inpatients (57.5% women) with chronic pain were examined (age between 16 and 79 years). The group as a whole yielded high scores on the somatisation dimension (SCL-90) and showed a considerable amount of psychopathological symptoms, such as depressive mood and anxiety (HADS) and a great emotional instability (FPI-R). Age differences were only found with regards to patients’ degree of aggression (SCl-90): younger patients showed higher aggressive tendencies than older ones (p< 0.05). The treatment offered helped patients in both age groups especially with regard to reduction of depressive mood (HADS, p< 0.01) and anxiety levels (HADS, p< 0.01). Regression analysis showed different age groups and gender as significant predictors of anxiety reduction under therapy (R²=.108; model: p< 0.01).

Discussion and conclusion

Results show that younger chronic pain patients suffer more from a considerable amount of psychological distress than older ones, but our treatment approach was equally effective in both groups. However, age and gender differences, as well as the patient’s baseline level of anxiety influenced the outcome. These factors need to be studied in future research.

     

A randomized, controlled trial of spinal endoscopic adhesiolysis in chronic refractory low back and lower extremity pain [ISRCTN 16558617]

Background

Postoperative epidural fibrosis may contribute to between 5% to 60% of the poor surgical outcomes following decompressive surgery. Correlations have been reported between epidural scarring and radicular pain, poor surgical outcomes, and a lack of any form of surgical treatment. The use of spinal endoscopic adhesiolysis in recent years in the management of chronic refractory low back and lower extremity pain has been described.

Methods

A prospective, randomized, double-blind trial was conducted to determine the outcome of spinal endoscopic adhesiolysis to reduce pain and improve function and psychological status in patients with chronic refractory low back and lower extremity pain. A total of 83 patients were evaluated, with 33 patients in Group I and 50 patients in Group II. Group I served as the control, with endoscopy into the sacral level without adhesiolysis, followed by injection of local anesthetic and steroid. Group II received spinal endoscopic adhesiolysis, followed by injection of local anesthetic and steroid.

Results

Among the 50 patients in the treatment group receiving spinal endoscopic adhesiolysis, significant improvement without adverse effects was shown in 80% at 3 months, 56% at 6 months, and 48% at 12 months. The control group showed improvement in 33% of the patients at one month and none thereafter. Based on the definition that less than 6 months of relief is considered short-term and longer than 6 months of relief is considered long-term, a significant number of patients obtained long-term relief with improvement in pain, functional status, and psychological status.

Conclusion

Spinal endoscopic adhesiolysis with targeted delivery of local anesthetic and steroid is an effective treatment in a significant number of patients with chronic low back and lower extremity pain without major adverse effects.     

The cubital tunnel syndrome caused by intraneural ganglion cyst of the ulnar nerve at the elbow: a case report

Background

Cubital tunnel syndrome is common nerve compression syndrome among peripheral nerve compression diseases. However, the syndrome caused by intraneural ganglion cysts has been rarely reported. Medical approaches, like ultrasound-guided aspiration and open surgical treatment remain to be discussed.

Case presentation

A 57-year-old woman presented with occasional pain, numbness and paralysis in her left hand and a palpable, painless mass in the ulnar side of her left elbow. Ultrasound-guided aspiration of the mass was performed to decompress the ulnar nerve. The patient experienced an evident release of pain in her hand, but symptoms of numbness and paralysis recurred 3 months later which greatly bothered the patient’s daily life. After evaluation, we had to perform an open surgery to excise the cyst. External neurolysis and anterior subcutaneous transposition were done. The patient was followed up for 2 years, and she made a complete recovery with no functional limitation.

Conclusions

The symptoms caused by intraneural ganglion cyst can be alleviated by accurate puncture. But puncture may be not complete and symptoms could recur. Complete external neurolysis can be counted as a complete and reliable treatment. Therefore, early diagnosis, careful preoperative imaging assessment and full decompression can be expected to receive a good rehabilitation.

     

Illness perception among patients with chest pain and palpitations before and after negative cardiac evaluation

Background

Patients with chest pain or palpitations often have poor outcomes following a negative cardiac evaluation, with symptom persistence, limitations in everyday activities, and reduced health-related quality of life. The aims of this study were to evaluate illness perceptions before and after negative cardiac evaluations and measure the ability of a self-report questionnaire to predict outcomes.

Methods

Patients (N?=?138) referred for chest pain or palpitations to a cardiac outpatient clinic were assessed before and six months after a negative cardiac evaluation. In addition to Brief Illness Perception Questionnaire (BIPQ), all patients completed the Beck Depression Inventory and SF-36 Health Survey.

Results

The emotional reactions to and understanding of symptoms had not improved six months after a negative cardiac evaluation. A stronger correlation between illness perceptions and health at follow-up than before the cardiac evaluation might explain the tendency for poor outcomes among these patients. Most of the eight BIPQ item scores before the negative cardiac evaluation were predictive of the outcome six months later. A single question asking about the perceived consequences of the complaints (BIPQ Item 1) rated before the cardiac evaluation was collapsed into a dichotomous variable with a cut-off at ≥4 which yields a sensitivity of 51%, a specificity of 85%, a positive predictive value of 71%, a negative predictive value of 69%, and an odds ratio of 5.7 (r?=?.38, p?<?.001) in predicting poor outcomes.

Conclusions

Assessing illness perceptions is important in patients with negative cardiac tests for understanding and predicting outcomes.

     

The beneficial use of ultramicronized palmitoylethanolamide as add-on therapy to Tapentadol in the treatment of low back pain: a pilot study comparing prospective and retrospective observational arms

Background

This pilot study was designed to compare the efficacy of ultramicronized palmitoylethanolamide (um-PEA) as add-on therapy to tapentadol (TP) with TP therapy only in patients suffering from chronic low back pain (LBP).

Methods

This pilot observational study consists in two arms: the prospective arm and the retrospective one. In the prospective arm patients consecutively selected received um-PEA as add-on therapy to TP for 6 months; in the retrospective arm patients were treated with TP only for 6 months. Pain intensity and neuropathic component were evaluated at baseline, during and after 6 months. The degree of disability and TP dosage assumption were evaluated at baseline and after 6 months.

Results

Statistical analysis performed with generalized linear mixed model on 55 patients (30 in the prospective group and 25 in the retrospective group) demonstrated that um-PEA as add-on treatment to TP in patients with chronic LBP, in comparison to TP alone, led to a significantly higher reduction in pain intensity, in the neuropathic component, the degree of disability and TP dosage assumption. No serious side effects were observed.

Conclusion

Overall, the present findings suggest that um-PEA may be an innovative therapeutic intervention as add-on therapy to TP for the management of chronic LBP with a neuropathic component, as well as to improve patient quality of life. Additionally, this combination treatment allowed a reduction in TP dose over time and did not show any serious side effects.

     

GRACE and TIMI risk scores but not stress imaging predict long-term cardiovascular follow-up in patients with chest pain after a rule-out protocol

Objective

To determine the long-term prognostic value of stress imaging and clinical risk scoring for cardiovascular mortality in chest pain patients after ruling out acute coronary syndrome (ACS).

Methods

A standard rule-out protocol was performed in emergency room patients with a normal or non-diagnostic admission electrocardiogram (ECG) within 6 h of chest pain onset. ACS patients were identified by troponin T, recurrent angina and serial ECG. Dobutamine stress echocardiography (DSE) was performed after ACS was ruled out. Myocardial perfusion scintigraphy (MPS) was performed within 6 months in an outpatient setting according to the physician’s discretion.

Results

524 patients were included. GRACE and TIMI risk scores were 75 (57–96) and 1 (0–2) in the rule-out ACS group, and 89 (74–107) and 2 (1–3) in the ACS group, respectively (median, interquartile range). Follow-up (median 9.4 (8.9–10.0) years) was complete in 96%. 350 of 379 rule-out ACS patients had an interpretable DSE and 52 patients underwent an MPS. 21 of the rule-out ACS patients (6%) died of a cardiovascular cause compared with 24 (17%) ACS patients (p?<?0.001). For rule-out ACS patients, C-statistics were 0.829 and 0.803 for the GRACE and TIMI scores. In these patients, DSE and MPS outcome did not predict long-term cardiovascular mortality. In multivariate analysis, known chronic heart failure, ACE inhibitor use, and GRACE score were independent predictors of cardiovascular mortality.

Conclusions

TIMI and GRACE score but not DSE and MPS are accurate predictors of long-term cardiovascular mortality, even in chest pain patients with a normal or non-diagnostic electrocardiogram undergoing a rule-out protocol.     

The Use of Physiotherapy among Patients with Subacromial Impingement Syndrome: Impact of Sex,Socio-Demographic and Clinical Factors

Background

Physiotherapy with exercises is generally recommended in the treatment of patients with subacromial impingement syndrome (SIS).

Objective

We aimed to investigate the use of physiotherapy in patients with SIS in Danish hospital settings as part of initial non-surgical treatment and after SIS-related surgery and to evaluate to which extent sex, socio-demographic and clinical factors predict the use of physiotherapy.

Methods

Using national health registers, we identified 57,311 patients who had a first hospital contact with a diagnosis of ICD-10, groups M75.1–75.9, 1 July 2007 to 30 June 2011. Records of physiotherapy were extracted within 52 weeks after first contact (or until surgery), and for surgically treated patients within 26 weeks after surgery. Predictors of the use of physiotherapy after first contact and after surgery were analysed as time-to-event.

Results

Within 52 weeks after first contact, 43% of the patients had physiotherapy and 30% underwent surgery. Within 26 weeks after surgery, 80% had a record of physiotherapy. After first contact and after surgery, exercise was part of physiotherapy in 65% and 84% of the patients, respectively. A public hospital contact, physiotherapy before hospital contact, administrative region, female sex, a diagnosis of other or unspecified disorders (M75.8-M75.9), and surgical procedure predicted higher use of physiotherapy. Low education level predicted slightly lower use of physiotherapy after first contact, but not after surgery.

Conclusion

In patients with SIS in Danish hospital settings, physiotherapy was more often used after surgery than as part of initial non-surgical treatment. The use of physiotherapy was less common among men than women, whereas unequal use of physiotherapy in relation to education level was not noticeable. The use of physiotherapy with exercises in initial non-surgical treatment was relatively limited.     

Addition of Lidocaine Injection Immediately before Physiotherapy for Frozen Shoulder: A Randomized Controlled Trial

The intraarticular injection of lidocaine immediately before a physiotherapy session may relieve pain during the stretching and mobilization of the affected joint in patients with a frozen shoulder, thus enhancing the treatment effect. To compare the effects of intraarticular injection of lidocaine plus physiotherapy to that of physiotherapy alone in the treatment of a frozen shoulder, a prospective randomized controlled trial was conducted in the rehabilitation department of a private teaching hospital. Patients with a frozen shoulder were randomized into the physiotherapy group or the lidocaine injection plus physiotherapy (INJPT) group. The subjects in the INJPT group underwent injection of 3 ml of 1% lidocaine into the affected shoulder 10 to 20 minutes before each physiotherapy session. In each group, the treatment lasted 3 months. The primary outcome measures were the active and passive range of motion of the affected shoulder. The secondary outcome measures were the results of the Shoulder Disability Questionnaire, the Shoulder Pain and Disability Index, and the 36-item Short-Form Health Survey (SF-36). The outcome measures were evaluated before treatment and 1, 2, 3, 4, and 6 months after the start of treatment. The group comparisons showed significantly greater improvement in the INJPT group, mainly in active and passive shoulder range of motion in flexion and external rotation and improvements in pain and disability (P < 0.05); however, no significant group difference was seen in the SF-36 results. The intraarticular injection of lidocaine immediately before a physiotherapy session might be superior to physiotherapy alone in the treatment of a frozen shoulder.

Trial Registration

ClinicalTrials.gov {"type":"clinical-trial","attrs":{"text":"NCT01817348","term_id":"NCT01817348"}}NCT01817348     

Disc degeneration implies low back pain

Background

Low back pain exerts a tremendous burden on individual patients and society due to its prevalence and ability to cause long-term disability. Contemporary treatment and prevention efforts are stymied by the absence of a confirmed cause for the majority of low back pain patients.

Methods

A system dynamics approach is used to build a physiologically-based model investigating the relationship between disc degeneration and low back pain. The model’s predictions are evaluated under two different types of study designs and compared with established observations on low back pain.

Results

A three-compartment model (no disc degeneration, disc degeneration with pain remission, disc degeneration with pain recurrence) accurately predicts the age-specific prevalence observed in one of the largest population-based surveys (R ²?=?0.998). The estimated transition age at which intervertebral discs lose the growth potential and begin degenerating is 13.3 years. The estimated disc degeneration rate is 0.0344/year. Without any additional change being made to parameter’s values, the model also fully accounts for the age-specific prevalence of disc degeneration detected with a lumbar MRI among asymptomatic individuals (R ²?=?0.978).

Conclusions

Dual testing of the proposed mechanistic model with two independent data sources (one with lumbar MRI and the other without) confirm that disc degeneration is the driving force behind and cause of age dependence in low back pain. Observed complexity of low back pain epidemiology arises from the slow dynamics of disc degeneration coupled with the fast dynamics of disease recurrence.

     

A child with anorexia nervosa presenting with severe infection with cytopenia and hemophagocytosis: a case report

Background

Patients with anorexia nervosa in the acute phase have physical complications, such as infectious disease. Although hemophagocytic syndrome due to infection is a rare complication in anorexia nervosa, early identification for hemophagocytosis is important for avoiding a life-threatening condition.

Case presentation

We report a case of a 12-year-old girl with anorexia nervosa presenting with infection with cytopenia and hemophagocytosis during initial nutritional therapy. She developed pyrexia, abdominal pain, and diarrhea during inpatient treatment. Although intravenous antibiotics were administered, the symptoms persisted. Acinetobacter baumannii was detected in blood culture. Hemophagocytosis was present in the bone marrow. Gamma globulin therapy was effective, with improvement in symptoms and cytopenia.

Conclusions

Although our case did not fulfill the criteria of hemophagocytic syndrome, clinicians should consider severe infection in anorexia nervosa with cytopenia and hemophagocytosis.

     

Anti-BCMA CAR-T cells for treatment of plasma cell dyscrasia: case report on POEMS syndrome and multiple myeloma

Background

POEMS (polyneuropathy, organomegaly, endocrinopathy, monoclonal gammopathy, and skin changes) syndrome still has no standard treatment. On the basis that both POEMS syndrome and myeloma have an underlying plasma cell dyscrasia, anti-myeloma therapy can be expected to be useful for POEMS syndrome. Chimeric antigen receptor T (CAR-T) cells targeting B cell maturation antigen (BCMA) has been used in the treatment of relapsed and refractory multiple myeloma (RRMM). No POEMS syndrome cases treated with anti-BCMA CAR-T cells have been reported.

Case presentation

Here, we, for the first time, report a POEMS syndrome case treated with anti-BCMA CAR-T cells. A 49-year-old female with incapacitating POEMS syndrome that progressed on lenalidomide treatment was enrolled in a phase I study involving anti-BCMA CAR-T cells (ChiCTR-OPC-16009113). Another patient with RRMM who had undergone six prior lines treatments was also enrolled in the study. They received infusions of anti-BCMA CAR-T cells. Both patients achieved a stringent complete response. Complete remission persisted in the patient with POEMS syndrome and lasted for 7.6 months before a relapse in RRMM patient. Both patients had toxicity consistent with the grade 1 cytokine release syndrome.

Conclusions

This is the first report of treatment by anti-BCMA CAR-T cells in POEMS syndrome. Our findings demonstrate the anti-BCMA CAR-T cell treatment may be a feasible therapeutic option for patients with POEMS syndrome and RRMM who do not respond well to traditional therapies.

Trial registration

ChiCTR-OPC, ChiCTR-OPC-16009113. Registered 29 August 2016.

     

Evaluation of circulating sRAGE in osteoporosis according to BMI,adipokines and fracture risk: a pilot observational study

Background

Osteoporosis is a systemic metabolic disease based on age-dependent imbalance between the rates of bone formation and bone resorption. Recent studies on the pathogenesis of this disease identified that bone remodelling impairment, at the base of osteoporotic bone fragility, could be related to protein glycation, in association to oxidative stress. The glycation reactions lead to the generation of glycation end products (AGEs) which, in turn, accumulates into bone, where they binds to the receptor for AGE (RAGE). The aim of this study is to investigate the potential role of circulating sRAGE in osteoporosis, in particular evaluating the correlation of sRAGE with the fracture risk, in association with bone mineral density, the fracture risk marker FGF23, and lipid metabolism.

Results

Circulating level of soluble RAGE correlate with osteopenia and osteoporosis level. Serum sRAGE resulted clearly associated on the one hand to bone fragility and, on the other hand, with BMI and leptin. sRAGE is particularly informative because serum sRAGE is able to provide, as a single marker, information about both the aspects of osteoporotic disease, represented by bone fragility and lipid metabolism.

Conclusions

The measure serum level of sRAGE could have a potential diagnostic role in the monitoring of osteoporosis progression, in particular in the evaluation of fracture risk, starting from the prevention and screening stage, to the osteopenic level to osteoporosis.