The Discrepancy between Patient and Clinician Reported Function in Extremity Bone Metastases

Background:. The Musculoskeletal Tumor Society (MSTS) scoring system measures function and is commonly used but criticized because it was developed to be completed by the clinician and not by the patient. We therefore evaluated if there is a difference between patient and clinician reported function using the MSTS score. Methods. 128 patients with bone metastasis of the lower (n = 100) and upper (n = 28) extremity completed the MSTS score. The MSTS score consists of six domains, scored on a 0 to 5 scale and transformed into an overall score ranging from 0 to 100% with a higher score indicating better function. The MSTS score was also derived from clinicians' reports in the medical record. Results. The median age was 63 years (interquartile range [IQR]: 55–71) and the study included 74 (58%) women. We found that the clinicians' MSTS score (median: 65, IQR: 49–83) overestimated the function as compared to the patient perceived score (median: 57, IQR: 40–70) by 8 points (p < 0.001). Conclusion. Clinician reports overestimate function as compared to the patient perceived score. This is important for acknowledging when informing patients about the expected outcome of treatment and for understanding patients' perceptions

Expert consensus document:Cholangiocarcinoma: current knowledge and future perspectives consensus statement from the European Network for the Study of Cholangiocarcinoma (ENS-CCA)

Cholangiocarcinoma (CCA) is a heterogeneous group of malignancies with features of biliary tract differentiation. CCA is the second most common primary liver tumour and the incidence is increasing worldwide. CCA has high mortality owing to its aggressiveness, late diagnosis and refractory nature. In May 2015, the "European Network for the Study of Cholangiocarcinoma" (ENS-CCA: www.enscca.org or www.cholangiocarcinoma.eu) was created to promote and boost international research collaboration on the study of CCA at basic, translational and clinical level. In this Consensus Statement, we aim to provide valuable information on classifications, pathological features, risk factors, cells of origin, genetic and epigenetic modifications and current therapies available for this cancer. Moreover, future directions on basic and clinical investigations and plans for the ENS-CCA are highlighted

Validation of the Spine Oncology Study Group—Outcomes Questionnaire to assess quality of life in patients with metastatic spine disease

General questionnaires are often used to assess quality of life in patients with spine metastases, although a disease-specific survey did not exist until recently. The Spine Oncology Study Group has developed an outcomes questionnaire (SOSG-OQ) to measure quality of life in these patients. However, a scoring system was not developed, and the questionnaire was not validated in a group of patients, nor was it compared with other general quality of life questionnaires such as the EuroQol 5 Dimensions (EQ-5D) questionnaire. Our primary null hypothesis is that there is no association between the SOSG-OQ and EQ-5D. Our secondary null hypothesis is that there is no difference in coverage and internal consistency between the SOSG-OQ and EQ-5D. We also assess coverage, consistency, and validity of the domains within the SOSG-OQ. A survey study from a tertiary care spine referral center was used for this study. The patient sample consisted of 82 patients with spine metastases, myeloma, or lymphoma. The SOSG-OQ (27 questions, 6 domains) score ranges from 0 to 80, with a higher score indicating worse quality of life. The EQ-5D (5 questions, 5 domains) index score ranges from 0 to 1, with a higher score indicating better quality of life. The association between the SOSG-OQ and EQ-5D index score was assessed using the Spearman rank correlation. Instrument coverage and precision were assessed by determining item completion rate, median score with range, and floor and ceiling effect. Internal consistency was assessed using Cronbach alpha. Multitrait analysis and exploratory factor analysis were used to analyze properties of the individual domains in the SOSG-OQ. The Spearman rank correlation between the SOSG-OQ and EQ-5D questionnaire was high (r=−0.83, p<.001). Internal consistency of the SOSG-OQ (0.92, 95% CI: 0.89–0.94) was higher as compared to the internal consistency of the EQ-5D (0.73, 95% CI: 0.63–0.84; p<.001). The SOSG-OQ score had no floor or ceiling effect indicating good coverage (median 30, range 3–64), whereas the EQ-5D had a ceiling effect of 10% (median 0.71, range 0.05–1). In conclusion, our study proposes a scoring methodology—after reversing four inversely scored items—for the SOSG-OQ and shows that the questionnaire is a valid tool for the assessment of quality of life in patients with metastatic spine disease. The SOSG-OQ is superior to the EQ-5D in terms of coverage and internal consistency but consists of more questions

Clinical characteristics and treatment outcomes in six cases of malignant tenosynovial giant cell tumor: initial experience of molecularly targeted therapy

Abstract Background Although tenosynovial giant cell tumor (TGCT) is classified as a benign tumor, it may undergo malignant transformation and metastasize in extremely rare occasions. High aberrant expression of CSF1 has been implicated in the development of TGCT and recent studies have shown promising activity of several CSF1R inhibitors against benign diffuse-type TGCT; however, little is known about their effects in malignant TGCT. Case presentation Information from six consenting patients (3 men, 3 women) with malignant TGCT presenting to Dana-Farber Cancer Institute for initial or subsequent consultation was collected. Median age at initial diagnosis of TGCT was 49.5 years (range 12–55), and median age at diagnosis of malignant TGCT was 50 years (range 34–55). Two patients developed malignant TGCT de novo, while four other cases showed metachronous malignant transformation. All tumors arose in the lower extremities (3 knee, 2 thigh, 1 hip). Five patients underwent surgery for the primary tumors, and four developed local recurrence. All six patients developed lung metastases, and four of five evaluable tumors developed inguinal and pelvic lymph node metastases. All six patients received systemic therapy. Five patients were treated with at least one tyrosine kinase inhibitor with inhibitory activity against CSF1R; however, only one patient showed clinical benefit (SD or PR). Five patients were treated with conventional cytotoxic agents. Doxorubicin-based treatment showed clinical benefit in all four evaluable patients, and gemcitabine/docetaxel showed clinical benefit in two patients. All six patients died of disease after a median of 21.5 months from diagnosis of malignant TGCT. Conclusions This study confirms that TGCT may transform into an aggressive malignant tumor. Lymph node and pulmonary metastases are common. Local recurrence rates are exceedingly high. Conventional cytotoxic chemotherapy showed clinical benefit, whereas tyrosine kinase inhibitors against CSF1R showed limited activity. Given its rarity, a prospective registry of malignant TGCT patients is needed to further understand the entity and to develop effective strategies for systemic treatment

Are Allogeneic Blood Transfusions Associated With Decreased Survival After Surgery for Long-bone Metastatic Fractures?

BACKGROUND: Previous studies have shown that perioperative blood transfusion increases cancer recurrence and decreases patient survival after resection of primary malignancies. The question arises whether this association also exists in patients with already disseminated disease undergoing surgery for metastatic long-bone fractures. PURPOSES: We sought to determine whether perioperative allogeneic blood transfusion is associated with decreased survival after operative treatment of long-bone metastatic fractures after accounting for clinical, laboratory, and treatment factors. Secondarily, we aimed to identify potential factors that are associated with decreased survival. METHODS: We included 789 patients in our retrospective study who underwent surgery at two institutions for a pathologic or impending metastatic long-bone fracture. We used multivariable Cox proportional hazards regression model analysis to assess the relationship of perioperative allogeneic blood transfusion with survival, and accounted for patient age, sex, comorbidities, BMI, tumor type, fracture type and location, presence of other bone and visceral metastases, previous radiotherapy and systemic therapy, preoperative embolization, preoperative hemoglobin level, treatment type, anesthesia time, blood loss, duration of hospital admission, year of surgery, and hospital. RESULTS: Considering transfusion as an “exposure,” and comparing patients who received transfusions with those who did not, we found that blood transfusion was not associated with decreased survival after accounting for all explanatory variables (hazard ratio [HR] 1.06; 95% CI, 0.87–1.30; p = 0.57). Evaluating transfusion in terms of dose-response, we found that patients who received more transfusions had lower survival compared with those who had fewer transfusions after accounting for all explanatory variables (HR per unit of blood transfused, 1.07; 95% CI, 1.02–1.12; p = 0.005). We found that age (HR, 1.02; 95% CI, 1.01–1.02; p < 0.001), comorbidity status (HR, 1.06; 95% CI, 1.01–1.10; p = 0.014), duration of hospital stay (HR, 1.02; 95% CI 1.00–1.03; p = 0.021), tumor type (HR, 1.71; 95% CI, 1.44–2.03; p < 0.001), and visceral metastases (HR, 1.59; 95% CI, 1.34–1.88; p < 0.001) were independently associated with survival. CONCLUSION: We found that exposure to perioperative allogeneic blood transfusion does not decrease survival, with the numbers available. However, our sample size might have been insufficient to reveal a small but potentially relevant effect. Our results do suggest a dose-response relationship; patients who received more transfusions had lower survival compared with those with fewer transfusions. Risk of death increased by 7% per unit of blood transfused. LEVEL OF EVIDENCE: Level III, prognostic study. ELECTRONIC SUPPLEMENTARY MATERIAL: The online version of this article (doi:10.1007/s11999-015-4167-3) contains supplementary material, which is available to authorized users

Are allogeneic blood transfusions associated with decreased survival after surgical treatment for spinal metastases?

Perioperative allogeneic blood transfusions have been associated with decreased survival after surgical resection of primary and metastatic cancer. Studies investigating this association for patients undergoing resection of bone metastases are scarce and controversial. We assessed (1) whether exposure to perioperative allogeneic blood transfusions was associated with decreased survival after surgery for spinal metastases and (2) if there was a dose-response relationship per unit of blood transfused. Additionally, we explored the risk factors associated with survival after surgery for spinal metastases. This is a retrospective cohort study from two university medical centers. There were 649 patients who had operative treatment for metastatic disease of the spine between 2002 and 2014. Patients with lymphoma or multiple myeloma were also included. We excluded patients with a revision procedure, kyphoplasty, vertebroplasty, and radiosurgery alone. The outcome measure was survival after surgery. The date of death was obtained from the Social Security Death Index and medical charts. Blood transfusions within 7 days before and 7 days after surgery were considered perioperative. A multivariate Cox proportional hazard model was used to assess the relationship between allogeneic blood transfusion as exposure versus non-exposure, and subsequently as continuous value; we accounted for clinical, laboratory, and treatment factors. Four hundred fifty-three (70%) patients received perioperative blood transfusions, and the median number of units transfused was 3 (interquartile range: 2-6). Exposure to perioperative blood transfusion was not associated with decreased survival after accounting for all explanatory variables (hazard ratio [HR]: 1.03; 95% confidence interval [CI]: 0.80-1.31; p=.841). Neither did we find a dose-response relationship (HR: 1.01; 95% CI: 0.98-1.04; p=.420). Other factors associated with worse survival were older age, more severe comorbidity status, lower preoperativehemoglobin level, higher white blood cell count, higher calcium level, primary tumor type, previous systemic therapy, poor performance status, presence of lung, liver, or brain metastasis, and surgical approach. Perioperative allogeneic blood transfusions were not associated with decreased survival after surgery for spinal metastases. More liberal transfusion policies might be warranted for patients undergoing surgery for spinal metastasis, although careful consideration is needed as other complications may occur

Thirty-day Postoperative Complications After Surgery For Metastatic Long Bone Disease Are Associated With Higher Mortality at 1 Year

BACKGROUND: The benefits of surgical treatment of a metastasis of the extremities may be offset by drawbacks such as potential postoperative complications. For this group of patients, the primary goal of surgery is to improve quality of life in a palliative setting. A better comprehension of factors associated with complications and the impact of postoperative complications on mortality may prevent negative outcomes and help surgeons in surgical decision-making. QUESTIONS/PURPOSES: (1) What is the risk of 30-day postoperative complications after surgical treatment of osseous metastatic disease of the extremities? (2) What predisposing factors are associated with a higher risk of 30-day complications? (3) Are minor and major 30-day complications associated with higher mortality at 1 year? METHODS: Between 1999 and 2016, 1090 patients with osseous metastatic disease of the long bones treated surgically at our institution were retrospectively included in the study. Surgery included intramedullary nailing (58%), endoprosthetic reconstruction (22%), plate-screw fixation (14%), dynamic hip screw fixation (2%), and combined approaches (4%). Surgery was performed if patients were deemed healthy enough to proceed to surgery and wished to undergo surgery. All data were retrieved by manually reviewing patients' records. The overall frequency of complications, which were defined using the Clavien-Dindo classification system, was calculated. We did not include Grade I complications as postoperative complications and complications were divided into minor (Grade II) and major (Grades III-V) complications. A multivariate logistic regression analysis was used to identify factors associated with 30-day postoperative complications. A Cox regression analysis was used to assess the association between postoperative complications and overall survival. RESULTS: Overall, 31% of the patients (333 of 1090) had a postoperative complication within 30 days. The following factors were independently associated with 30-day postoperative complications: rapidly growing primary tumors classified according to the modified Katagiri classification (odds ratio 1.6; 95% confidence interval, 1.1-2.2; p = 0.011), multiple bone metastases (OR 1.6; 95% CI, 1.1-2.3; p = 0.008), pathologic fracture (OR 1.5; 95% CI, 1.1-2.0; p = 0.010), lower-extremity location (OR 2.2; 95% CI, 1.6-3.2; p < 0.001), hypoalbuminemia (OR 1.7; 95% CI, 1.2-2.4; p = 0.002), hyponatremia (OR 1.5; 95% CI, 1.0-2.2; p = 0.044), and elevated white blood cell count (OR 1.6; 95% CI, 1.1-2.4; p = 0.007). Minor and major postoperative complications within 30 days after surgery were both associated with greater 1-year mortality (hazard ratio 1.6; 95% CI, 1.3-1.8; p < 0.001 and HR 3.4; 95% CI, 2.8-4.2, respectively; p < 0.001). CONCLUSION: Patients with metastatic disease in the long bones are vulnerable to postoperative adverse events. When selecting patients for surgery, surgeons should carefully assess a patient's cancer status, and several preoperative laboratory values should be part of the standard work-up before surgery. Furthermore, 30-day postoperative complications decrease survival within 1 year after surgery. Therefore, patients at a high risk of having postoperative complications are less likely to profit from surgery and should be considered for nonoperative treatment or be monitored closely after surgery. LEVEL OF EVIDENCE: Level III, therapeutic study

Development of a Prognostic Survival Algorithm for Patients with Metastatic Spine Disease

Background: Current prognostication models for survival estimation in patients with metastatic spine disease lack accuracy. Identifying new risk factors could improve existing models. We assessed factors associated with survival in patients surgically treated for spine metastases, created a classic scoring algorithm, nomogram, and boosting algorithm, and tested the predictive accuracy of the three created algorithms at estimating survival. Methods: We included 649 patients from two tertiary care referral centers in this retrospective study (2002 to 2014). A multivariate Cox model was used to identify factors independently associated with survival. We created a classic scoring system, a nomogram, and a boosting (i.e., machine learning) algorithm and calculated their accuracy by receiver operating characteristic analysis. Results: Older age (hazard ratio [HR], 1.01; p = 0.009), poor performance status (HR, 1.54; p = 0.001), primary cancer type (HR, 1.68; p 1 spine metastasis (HR, 1.32; p = 0.009), lung and/or liver metastasis (HR, 1.35; p = 0.005), brain metastasis (HR, 1.90; p < 0.001), any systemic therapy for cancer prior to a surgical procedure (e.g., chemotherapy, immunotherapy, hormone therapy) (HR, 1.65; p < 0.001), higher white blood-cell count (HR, 1.03; p = 0.002), and lower hemoglobin levels (HR, 0.92; p = 0.009) were independently associated with decreased survival. The boosting algorithm was best at predicting survival on the training data sets (p < 0.001); the nomogram was more reliable at estimating survival on the test data sets, with an accuracy of 0.75 (30 days), 0.73 (90 days), and 0.75 (365 days). Conclusions: We identified risk factors associated with survival that should be considered in prognostication. Performance of the boosting algorithm and nomogram were comparable on the testing data sets. However, the nomogram is easier to apply and therefore more useful to aid surgical decision-making

Complications after surgery for metastatic humeral lesions

Background: Knowledge of surgical outcome and its predictors helps inform patients and aids in surgical decision-making. We aimed to assess the outcome-reoperation and systemic complication rate-of surgery for humeral metastases, myeloma, or lymphoma. Our null hypothesis was that there are no factors associated with these outcomes. Methods: We included 295 consecutive patients in this retrospective study: 134 (45%) proximal, 131 (44%) diaphyseal, and 30 (10%) distal impending or pathologic fractures. Proximal lesions were treated by intramedullary nailing (43%, n = 57), prosthesis (34%, n = 46), plate-screw fixation (22%, n = 30), and a combination (n = 1). Diaphyseal lesions were treated by intramedullary nailing (69%, n = 91), plate-screw fixation (30%, n = 39), and a combination (n = 1). Distal lesions were treated by platescrew fixation (97%, n = 29) and intramedullary nailing (3.3%, n = 1). Results: We found 25 (8.5%) reoperations, and 17 (5.8%) patients had 18 systemic complications: pneumonia (3.7%, n = 11), pulmonary embolism(1.3%, n = 4), sepsis (0.68%, n = 2), and fat embolism(0.34%, n = 1). No factors were independently associated with reoperation. Logistic regression analysis demonstrated that favorable cancer status (i.e., a higher modified Bauer score: odds ratio, 0.48; 95% confidence interval, 0.29-0.80; P =.005) was independently associated with a decreased systemic complication rate. Conclusion: Poor cancer status was an independent predictor of postoperative systemic complications. This could help inform the patient and anticipate postoperative problems. (C) 2016 Journal of Shoulder and Elbow Surgery Board of Trustees