scholarly journals Role of patients associations in connective tissue calcifiying diseases: a position statement from EuroSoftCalc.Net group

2021 ◽  
Vol 16 (1) ◽  
Author(s):  
Pedro Valdivielso ◽  
Marta Jacinto ◽  
Guillemette Devernois ◽  
Jorge Laplana ◽  
Maria García-Fernández ◽  
...  

AbstractPatients have been showing a growing interest in taking active participation in decision making, and having the opportunity to drive clinical investigation. This is more common for patients who have a rare disease than for those with more prevalent diseases. The EuroSoftCalc.Net COST Action, a group of clinicians and researchers involved in the dystrophic calcification process held a meeting in which three representatives of patients’ associations, coming from Portugal, France and Spain, discussed the role of patients and their associations, namely in the Action, and also the main concerns in their countries. The disparities in health care between European Union countries with regard to connective tissue calcifying diseases, and the existing conflicts of interest, were a matter of debate during the meeting. As a consequence of the presentations and the debate that followed, it became clear that, despite their countries, the main concerns of the patients are identical, namely a lack of specific therapy and follow-up clinical guidelines, delays in the diagnosis, difficulties in getting members to enrol to associations, and/or difficulties with doctors’ explanations for the diseases. The attendees also agreed that EuroSoftCalc.Net group should help to set up new associations where no Patient Associations presently exist, and, furthermore, should release diagnosis and follow-up guidelines, especially helpful in countries, and/or for diseases, where no multidisciplinary consultations are available.

2020 ◽  
Vol 6 (3) ◽  
pp. 89-91
Author(s):  
Christina Hari Nawangsih Prihharsanti ◽  
Muhamad Rizqi Setyarto ◽  
Dion Firli Bramantyo

Background: Keloid is a benign growth of connective tissue. There are several risk factors that play a role in keloid growth. Excision surgery is one of the modalities in the treatment of keloids. However, excision surgery alone has a recurrence rate of 45-100%.Case: Male, 37 years old, with complaints arising from a keloid lesions in the left earlobe since three years ago with a history of previous injuries. The lesions is then operated on but always grows back postoperatively. The number of surgeries that have been carried out three times with further treatment in the form of corticosteroid injection. However, keloid still recurrence. Finally it was decided to undergo treatment with surgery followed by radiotherapy within a period of no more than 24 hours postoperatively. Follow-up after six months gave good results without recurrence.Discussion: Keloid has a high recurrence rate after excision surgery. Surgery followed by radiotherapy has a low recurrence rate compared to surgery or surgery followed by administration of corticosteroid injections. Radiotherapy as adjuvant therapy for postoperative keloid excision has the role of sterilizing the connective tissue stem cell active fibroblasts and acute inflammatory cells that grow in the early postoperative period. A study states that administration of postoperative radiation with electrons in keloids in the earlobe at a dose of 15 Gy in three fractions gives a low recurrence rate and a low risk of side effects in the surrounding soft tissue. 


Blood ◽  
2011 ◽  
Vol 118 (21) ◽  
pp. 3182-3182
Author(s):  
Srila Gopal ◽  
Rao Madhumathi ◽  
Damien R Ashby

Abstract Abstract 3182 Methods: We carried out a prospective study of 167 patients with CKD stages 1 to 4 followed over a two year period. Using a competitive radioimmunoassay, we measured plasma hepcidin at baseline and studied its association to baseline clinical parameters, as well as the development of anemia and need for ESA over a two year follow up period. Exclusion criteria included any erythropoietin stimulating agent (ESA) therapy at baseline. Variables were log transformed to satisfy normality assumptions. Results: Median (P5-P95) Glomerular filtration rate (GFR) was 42 (20–109) ml/min and 45% were anemic at the time of enrollment by KDOQI criteria, 11 % (18/167) having severe anemia. In patients with CKD, Hepcidin was positively correlated with serum iron (Spearmans rho,p:serum iron,0.22,0.005) and negatively correlated to EPO levels (Spearmans rho,p:-0.23,0.003 vss.-0.55,0.002). Hepcidin levels at baseline were not significantly associated with GFR, hemoglobin or CRP, and did not differ according to diabetes or ethnicity. Though Hepcidin levels did not differ by the presence of anemia at baseline, they were significantly higher among patients who developed severe anemia (n=23) at the end of the first year (40 ng/mL vs. 25 ng/mL, p<0.01). A total of 13 patients initiated ESA during follow up; a 2-fold higher hepcidin was associated with a hazard ratio of 2.25 (95% CI 0.89 to 5.62) for the need to initiate ESA therapy (Fig 1), adjusted for GFR. Conclusion: High hepcidin, unrelated to inflammation, is a predictor for the development of anemia and the need for initiation of ESA therapy in CKD patients. The role of Hepcidin as a therapeutic target in the anemia of CKD needs further study. Disclosures: No relevant conflicts of interest to declare.


2003 ◽  
Vol 212 ◽  
pp. 555-557
Author(s):  
Nicole L. Homeier ◽  
Robert D. Blum ◽  
Peter S. Conti ◽  
Anna Pasquali ◽  
Augusto Damineli

Most of the Milky Way's evolved massive stellar population is hidden from view. We can attempt to remedy this situation with near-infrared observations, and in this paper we present our method for detecting Wolf-Rayet stars in highly extincted regions and apply it to the inner Galaxy. Using narrow-band filters at K-band wavelengths, we demonstrate how WR stars can be detected in regions where they are optically obscured. Candidates are selected for spectroscopic follow-up from our relative line and continuum photometry. The final results of applying this method with a near-IR survey in the Galactic plane will provide a more complete knowledge of the structure of the galactic disk, the role of metallicity in massive stellar evolution, and environments of massive star formation. In this paper we briefly describe the survey set-up and report on recent progress. We have discovered four emission-line objects in the inner Galaxy: two with nebular emission lines, and two new WR stars, both of late WC subtype.


Blood ◽  
2013 ◽  
Vol 122 (21) ◽  
pp. 4082-4082 ◽  
Author(s):  
Mario Tiribelli ◽  
Federico De Marchi ◽  
Daniela Barraco ◽  
Luciana Marin ◽  
Erika Codarin ◽  
...  

Abstract Introduction Evolution to myelofibrosis (MF) represents a relatively rare but always severe event in patients with essential thrombocythemia (ET) and polycythemia vera (PV). Few reports have focused on the clinical and biological features at diagnosis of ET and PV that correlate with progression to MF. Aims and Methods We retrospectively studied a series of patients with post-ET and post-PV MF and compared with a group of ET and PV patients with a long follow-up without myelofibrotic evolution, with the aim to identify prognostic factors for MF. Forty-three patients with post-ET (n=29) and post-PV (n=14) MF followed at our institution were compared with 125 ET and 75 PV patients with at least 9 years of follow-up without evolution. Diagnosis of ET and PV was confirmed according to WHO criteria (including JAK2 analysis, performed since 2006 and study), evolution to MF was defined according to IWG-MRT proposed criteria. The following parameters, available for all patients at diagnosis of ET or ET, were taken into consideration to find prognostic risk factors for myelofibrosis: age, platelet (PLT) count, hemoglobin (Hb) and hematocrit (Hct) levels, white blood cell (WBC) count. Statistical analyses were conducted using Student t test. Results Median time from diagnosis of ET/PV and progression to MF was 156 months (range: 29-314). Comparing baseline characteristics of patients who evolved to MF and those who did not, we did not found any significant correlation. Mean data at diagnosis for patients with (n = 43) or without (n=200) subsequent evolution to MF were as follow: age 52.1 vs 53.1 years (p=0.79), Hb 15.4 vs 15.7 g/dl (p=0.59), Hct 47.2 vs 47.1% (p=0.67), WBC 9.8 vs 9.1 x 109/l (p=0.11), PLT 713 vs 689 x 109/l (p=0.87). Also when considering only the 29 post-ET MF and the 125 ET patients, there was no clinical feature present at diagnosis that could foresee a future myelofibrotic evolution. Conversely, in the 14 post-PV MF and 75 PV patients, progression to MF was predicted only by higher WBC count (11.4 vs 9.3 x 109/l, p=0.046), while no correlation was found with age, Hb, Hct or PLT [Table 1]. Conclusions Concordant with some previous reports, our data suggest a possible role of leucocytosis as an adverse risk factor for progression to MF in patients with PV, though not in ET. Other clinical characteristics present at diagnosis, such as advanced age, anemia or polycythemia and thrombocytosis do not seem to be associated with higher risk of fibrotic evolution in patients with myeloproliferative neoplasms. Disclosures: No relevant conflicts of interest to declare.


Blood ◽  
2019 ◽  
Vol 134 (Supplement_1) ◽  
pp. 4186-4186
Author(s):  
Giovanni Caocci ◽  
Maria Pina Simula ◽  
Silvia Ghiani ◽  
Olga Mulas ◽  
Giorgia Mainas ◽  
...  

Background. Transfusion-associated iron overload may increase the risk of infections both increasing bacterial or fungal growth and leading to the production of free iron that impairs immune system response. Anemia and transfusion dependency (TD) represent well known prognostic factors of survival in patients with myelofibrosis (MF). Compared to myelodysplastic syndrome (MDS), the role of iron overload on infection in MF has been scarcely explored. Methods. We identified consecutive adult patients diagnosed at our Centre with primary or secondary MF, between 1998 and 2018. Patients were stratified in 2 groups according to transfusion dependence defined as having received >2 unit of red blood cells (RBC) over 3 months. The total number of RBC units infused was also collected. All infections were recorded and graded according to Common Terminology Criteria for Adverse Events (CTCAE). Results. A total of 106 patients, median age 72 years (range 44-89) were retrospectively evaluated. A diagnosis of primary MF was performed in 75 of cases (71%), post Essential Thrombocythemia myelofibrosis (PET-MF) in 27 (25%) and post Polycythemia Vera myelofibrosis (PPV-MF) in 4 (4%). Splenomegaly was present in 83 patients (78%) and 30 (28%) reported constitutional symptoms.According to the IPSS, 43 patients (40 %) were classified at low or intermediate-1 risk, and 63 (60 %) at intermediate-2 or high risk. Molecular analysis was performed in 76 patients (72%): 54 of 76 patients (71%) were positive for the JAK2V617F mutation, 10 (13%) for CALR mutation, 4 (5%) for MPL, and 8 (11%) were negative for all tested mutations. Over time, 56 patients (53%) received hydroxyurea or other cytoreductive treatment, and 23 (22%) ruxolitinib. Overall, 39 patients (37%) were transfusion dependent with a median of 14 RBC units received during follow-up (range 4-199). The median serum ferritin level in the TD cohort was 840 ng/mL (range 130-12.129). Median observation time was 36 months (range 3-203). At last follow-up, 48 patients (45%) presented one or more infectious episodes. Total infectious events were 69 and 13 of them (19%) were defined as severe (grade 3-4 CTCAE). Anatomical site of infections was the respiratory tract in 28 (41%) of cases, gastro-intestinal in 22 (32%), gynecological-urological in 8 (11%), sepsis in 2 (3%), unspecified in the remaining cases. When detected, the etiological agents were bacterial in 8 (12%), viral in 5 (7%) and fungal in 4 (6%). The 60-month cumulative incidence of infection from diagnosis of MF was 64.1±6.5%. TD patients showed a higher incidence of infection (99±8.8% vs 53.3±8%, p=0.007) (Figure 1). In multivariate analysis no association was found between infection incidence and primary vs secondary MF, splenomegaly, age >65 years, ruxolitinib treatment; only transfusion dependencemaintained a significant association (p=0.019; HR=2.13; 95% C.I.=1.13-4.01). Among the 39 TD patients, the transfusion burden was significantly higher in those with infectious complication (median 24 RBC units vs 15 RBC units; p=0.012). The 60-month overall survival was 40±5.9%. Lower IPSS-risk (p<0.0001) and ruxolitinib treatment (p=0.027) were significantly associated with higher survival. Conclusions. This retrospective monocentric real-life study showed an increased risk of infections in MF patients with higher transfusion burden. The role of iron chelation in improving infection free survival in patients with MF and iron overload should be further investigated. Figure 1 Disclosures No relevant conflicts of interest to declare.


Kardiologiia ◽  
2021 ◽  
Vol 61 (6) ◽  
pp. 28-34
Author(s):  
O. V. Kamenskaya ◽  
I. Yu. Loginova ◽  
A. S. Klinkova ◽  
V. V. Lomivorotov ◽  
A. M. Chernyavsky

Aim    To present clinical observations of the novel coronavirus infection (COVID-19) in patients with chronic thromboembolic pulmonary hypertension (CTEPH) after a surgical intervention in the form of thromobendarterectomy from pulmonary artery branches.Material and methods    The Acad. E.N. Meshalkin National Medical Research Center performed 127 open surgical interventions for CTEPH in the form of thromobendarterectomy from 2016 through 2020. The present study enrolled 113 patients included into the follow-up care group and into the Center Registry who were followed up for more than 6 months after the surgery. Clinical and functional features of COVID-19 were evaluated in the studied group.Results    In the follow-up care group, 5 (4.4%) postoperative CTEPH patients had COVID-19. One patient had asymptomatic disease, and others had typical clinical symptoms and bilateral polysegmental pneumonia. There were no cases requiring artificial ventilation and no lethal outcomes. All patients with COVID-19 received anticoagulants as a basis therapy for CTEPH, and two patients who had residual pulmonary arterial hypertension (PAH) additionally received a PAH-specific therapy. During the treatment of COVID-19, no adjustment of the anticoagulant or PAH-specific therapy was required.Conclusion    The group of patients with CTEPH is a unique pathophysiological model for studying the effect of COVID-19 under the conditions of compromised pulmonary circulation. In the studied follow-up care group, the COVID-19 morbidity was 4.4 % without fatal outcomes. Evaluation of the role of chronic anticoagulant and PAH-specific therapy in COVID-19 postoperative patients as well as evaluation of the role of COVID-19 in CTEPH progression merit further investigation. 


2012 ◽  
Vol 4 (2) ◽  
pp. 110-112 ◽  
Author(s):  
Deepti Shrivastava ◽  
Shikha Joshi ◽  
Hariharan Chella

ABSTRACT Introduction Puberty menorrhagia is a real trouble among adolescent girls mostly due to problem in hypothalamic-pituitaryovarian axis. Aims and objectives To understand the various causative factors in puberty menorrhagia and to evaluate the role of hormones estrogens, progesterones or combination of both in controlling the excessive menstrual bleeding. Materials and methods It is a study conducted in the Department of Obstetrics and Gynecology at Acharya Vinoba Bhave Rural Hospital, JNMC, Sawangi from January 2009 to December 2010. Study included a total of 50 adolescent girls presenting with complains of menorrhagia. Results About 78% patients had menorrhagia due to immaturity of hypothalamic-pituitary-ovarian axis; 14% had polycystic ovarian disease and 8% had hypothyroidism. Out of 50 patients who were treated with hormones, 26 patients (52%) responded by the end of third month follow-up. Conclusion Majority of the patients showed good response to combined oral contraceptive pills (78%). How to cite this article Joshi S, Chella H, Shrivastava D. Study of Puberty Menorrhagia in Adolescent Girl in a Rural Set-up. J South Asian Feder Obst Gynae 2012;4(2):110-112.


Blood ◽  
2009 ◽  
Vol 114 (22) ◽  
pp. 5007-5007
Author(s):  
Matteo Pelosini ◽  
Francesco Caracciolo ◽  
Sara Galimberti ◽  
Edoardo Benedetti ◽  
Federico Papineschi ◽  
...  

Abstract Abstract 5007 Introduction Non Hodgkin Lymphoma represent a category of hematological malignances which are chemo and radio-sensitive; improvements in their treatment had been achieved by immunotherapeutic approaches. However some patients will relapse after achieving complete remission (CR). Obviously, in order to detect and possibly treat them as soon as possible, a follow up strategy has to be planned. The more diffuse follow up have been planned years before the introduction of innovative methods and imaging techniques, suggesting the opportunity to revise these programs. In particulary it is not still clear which is best techniques useful to properly follow this patient. Recently new interesting methods are available like PET, CT-PET and minimal residual disease (MRD) monitoring. Methods 418 NHL patients -both low and high grade- treated at our institution from 1995 to 2005 who achieved a CR status according to Cheson criteria have been evaluated. LH NHL included follicular lymphoma, lymphoplasmocytic lymphoma, Marginal zone lymphoma, and small lymphocytes lymphoma. In the HG NHL, we included T-cells lymphomas, diffuse large cell lymphoma, lymphoblastic lymphoma, Mantle cell lymphoma, anaplastic lymphoma, Burkitt lymphoma. Patient characteristics are summarized in Table 1. Follow up is planned for 5 years divided in two periods: in the first two years patients are evaluated every 3 months and in the following three years every sixth month. At each visit physical examinations, blood testing (blood count, chemistry) are performed; for imaging techniques we alternate a whole body CT scans to ultrasounds and chest X-ray coupled. Bone marrow samples for both pathological and molecular analysis are collected every six months in the first period and once a year afterwards. PETs were usually performed when CT showed uncertain findings. Results There were 431 events, with 188 first relapses, 86 second, 18 third, 4 fourth and 1 fifth relapses. Relapse rate was similar among high and low grades, (37% and 35 % respectively) but time to relapse was longer for low grades (18.2 months vs 8.9 months). There was not relationship between IPI status and relapse rate. 72 % of relapse was at the same site of diagnosis. Relapses were detected by ultrasound in 139 cases (32 %), CT scans in 110 (25.5%) and by physical examination in 62 (14.4%). Remaining patients' relapse were diagnosed with other techniques (lab test, gastroscopy, NRM) New techniques as MRD monitoring, PET or PET/CT were not available for many patients, anyway MRD monitoring was able to detect disease re-appearance in 2%, and we had a total of 28 cases (6,5%) of relapse diagnosis with PET, but we noted a total of 18,5 % of false positive. Discussion and conclusions Many papers from literature raised many questions about which is the best techniques to follow patients. Many authors showed how symptoms onset and clinical findings appeared to be the more important for relapse detection compared to imaging before and during CT era. Some works pointed out also that even when CT detected earlier a relapse that do not translate in a survival advantage. Recently much interest has been focused on PET, CT-PET and MRD. They two appeared to be very important as prognostic tolls but their role for follow up purpose is still debatable. On the basis of clinical data and of these consideration routine PET is not recommended during follow up. Unfortunately PETs and MRD monitoring were not available for the majority of our patients, diagnosed in the nineteen's. In conclusion in our experience we observed some usefulness of CT scans and ultrasounds but we must recall that the majority of literature is not consistent with our results. Considering our experience and data from literature probably imaging should be performed routinely at the end of therapy, and during follow up only on the basis of presentation and clinical suspicion. As a matter of fact NCCN reviewed its guidelines do not suggesting a wide use of routine imaging. Further investigation by clinical and randomized trials are certainly needed to better understand, in particular the role of PET-PET/CT for follow up purpose. Disclosures No relevant conflicts of interest to declare.


Blood ◽  
2010 ◽  
Vol 116 (21) ◽  
pp. 4591-4591
Author(s):  
Bishoy Faltas ◽  
Jane L. Liesveld ◽  
Michael Becker ◽  
Jainulabdeen J. Ifthikharuddin ◽  
Gordon L. Phillips

Abstract Abstract 4591 Although the role of AHSCT1 is well established in multiple myeloma therapy, the role of the salvage (not “tandem”) AHSCT2 is less clear. However, most pts. undergo initial stem cell harvests with plans for eventual AHSCT2. To clarify the indications, the prognostic factors and the outcomes of AHSCT2 in relapsed myeloma, we analyzed our experience in 19 pts. (pts.) who underwent salvage AHSCT2 between the 1994–2008. Mean age at AHSCT1/2 was 54.58 (SD 7.96)/57.03 (SD 8.10) respectively; 15 (79.0%) were males. At the time of diagnosis, 7 (36.8%) pts. had ISS stage I, 6 (31.6%) stage II, 3(15.8%) stage III and in 3 (15.8%) the ISS stage could not be determined. Isotypes: IgG 11 (57.9%), IgA 4 (21.1%), 1 (5.3%) pt. had IgM and 3 (15.8%) had light chain myeloma. Cytogenetics were normal in 6 (31.6%), abnormal in 5 (26.3%) and unavailable for 8 (42.1%) pts. The most common therapy received prior to AHSCT1 was Vincristine/Adriamycin/Dexamethasone in 11 (57.9%) pts.; 7 pts. were exposed to novel agents. One pt. achieved complete response (CR), 15 (79.0%) pts. achieved partial response (PR) and 3 (15.8%) pts. had stable or progressive disease after receiving the initial treatment. All pts. proceeded to receive high dose therapy with Melphalan (MEL), 2 pts received fTBI in addition to MEL. Mean MEL dose was 190.00 mg/m2 (SD 23.01); 15 pts. received 200 mg/m2. All pts. received >2×10e6/kg of CD34+ cells in the first and second transplants. At D+100 after AHSCT1, responses were: 4 (21.1%) CR, 1 (5.3%) VGPR, 8 (42.1%) PR and 6 (31.6 %) with lesser responses. Therapy to prolong response or for salvage after AHSCT1 was given in all pts before AHSCT2, including Thalidomide in 6 (31.6%), Bortezomib in 4 (21.1%) and Lenalidomide in 2 (10.5) pts. Median time to progression after AHSCT1 was 318 days [95 % CI 110– 573]. Median interval between AHSCT1 and AHSCT2 was 896.74 days (SD: 698.34 days). At the time of AHSCT2, 4 (21.1%) were in PR, 15 (79.0%) had progressive disease. For AHSCT2, all pts. Received MEL, one pt. received MEL + Cytoxan and one pt. received MEL + Bortezomib. The median MEL dose/m2 was 175.56 (52.04).All pts. survived AHSCT2 to D+100, responses were as follows: 3 (15.8%) VGPR, 9 (47.4%) in PR; 7 (36.9) pts. had lesser responses. After AHSCT2, nine (47.4%) pts. had grade III toxicity and only one pt. had grade IV toxicity (avascular necrosis). Maintenance therapy after AHSCT2 included Bortezomib in 7 (36.8%) pts., Lenalidomide in 5 (26.3%) pts. and Thalidomide in 4 (21.1%) pts. After AHSCT2, the median overall survival (OS) was 658 days [95 % CI 326–1330] and progression free survival (PFS) after AHSCT2 was 237 days [95 % CI 121– 397] (Figure 1). OS probability at 6, 12, 24 months after AHSCT2 was 81.3, 75.0, 39.3 % respectively. For all pts., the median OS time (i.e. time from diagnosis to death or last follow-up) was 2187 days [95% CI 1413– 4126]. At the end of the follow up period, a total of 14 pts. had died. Causes of death were progression in 12 (63.2%) and sepsis in 2 pts. (10.5%). The number of previous lines of chemotherapy, interval between transplants, disease status at the time of AHSCT1/2, type of myeloma and MEL dose were not predictive of OS and PFS. In the multivariate analysis, only age by decade at AHSCT2 and male gender were independent predictors of OS after AHSCT2 (HR 4.037, P= 0.01), (HR 3.74, P=0.07) respectively. Similarly, in the multivariate analysis for PFS after AHSCT2, age at AHSCT2 was an unfavorable independent predictor (HR 3.48, P=0.009) whereas relapse free response more than 18 months after AHSCT1 was an independent favorable predictor (HR 0.198, P=0.007).Our results are consistent with the few studies examining the impact of salvage transplants for myeloma which report a median overall survival ranging from 20.7 to 38.1 months from the time of AHSCT2.We conclude that salvage AHSCT2 can positively impact PFS and OS but efforts to improve outcomes are mandatory.FigureSEQ Figure ≂,* ARABIC 1Figure. SEQ Figure ≂,* ARABIC 1 X-axis represents time from AHSCT2 in days. Y axis represents survival distribution. Blue line = OS, Red = PFS. Disclosures: No relevant conflicts of interest to declare.


2017 ◽  
Vol 30 (3) ◽  
pp. 307-317 ◽  
Author(s):  
Giorgina Barbara Piccoli ◽  
◽  
Gianfranca Cabiddu ◽  
Santina Castellino ◽  
Giuseppe Gernone ◽  
...  

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