Ultrasound evaluation of joint damage and disease activity in adult patients with severe haemophilia A using the HEAD‐US system

Haemophilia ◽  
2021 ◽  
Author(s):  
Víctor Jiménez‐Yuste ◽  
Hortensia de la Corte‐Rodríguez ◽  
María Teresa Álvarez‐Román ◽  
Mónica Martín‐Salces ◽  
Felipe Querol ◽  
...  
2008 ◽  
Vol 99 (01) ◽  
pp. 71-76 ◽  
Author(s):  
Karin Kurnik ◽  
Frauke Friedrichs ◽  
Susan Halimeh ◽  
Anne Krümpel ◽  
Christoph Bidlingmaier ◽  
...  

SummaryPatients with severe haemophilia A (HA) can either be treated by regular FVIII infusions twice or three times per week (prophylaxis), or only in case of bleeding episodes (on-demand). Whereas prophylaxis reduces the number of bleeding episodes and may therefore prevent the development of haemophilic arthropathy, there is still a lot of controversy surrounding recommendations on age and dose at start of prophylactic regimens. The present database study was performed to investigate the role of primary versus secondary prophylaxis in HA children. The outcome variable was imaging-proven haemophilic joint damage. Forty-two children were initially treated with primary prophylaxis following the first bleeding episode, and were frequency-matched (year of birth, catchment area) to 67 pa- tients receiving “on-demand” therapy with an early switch to “secondary prophylaxis”. In multivariate analysis adjusted for the HA mutation type and the presence or absence of thrombophilia, the Pettersson score investigated at a median age of 12.5 years in joints with at least one documented bleeding episode was not significantly different between the two patient groups (p=0.944),and no statistically significant differences were found in patients with target joints (p=0.3), nor in children in whom synovitis had occurred (p=0.77). No conclusion can be drawn from the data presented herein whether primary prophylaxis or an early start of secondary prophylaxis is superior with respect to joint outcome in children with severe HA.


2014 ◽  
Vol 134 ◽  
pp. S33-S37 ◽  
Author(s):  
Johannes Oldenburg ◽  
Hans-Hermann Brackmann

Haemophilia ◽  
2013 ◽  
Vol 19 (3) ◽  
pp. e167-e173 ◽  
Author(s):  
M. N. D. Di Minno ◽  
S. Iervolino ◽  
E. Soscia ◽  
A. Tosetto ◽  
A. Coppola ◽  
...  

Haemophilia ◽  
2021 ◽  
Author(s):  
Rachel Goren ◽  
Eleanor Pullenayegum ◽  
Victor S. Blanchette ◽  
Saunya Dover ◽  
Manuel Carcao ◽  
...  

Blood ◽  
2016 ◽  
Vol 128 (22) ◽  
pp. 3802-3802
Author(s):  
Juan Eduardo Megías-Vericat ◽  
María Remedios Marqués ◽  
Saturnino Haya ◽  
Ana Rosa Cid ◽  
Felipe Querol ◽  
...  

Abstract Background: Prophylaxis with recombinant factor VIII (rFVIII) is considered the optimal treatment for severe or moderate haemophilia A (HA). Bleeding into joints determines a proportional chronic joint damage to its frequency and severity. Knowledge of the individual's pharmacokinetics (PK) using Bayesian analysis helps to individualize prophylaxis therapy with recombinant factor VIII (rFVIII) in severe or moderate haemophilia A (HA) and minimize the risk of bleeding, extracting only 2-3 samples. Methods Retrospective study in HA patients with rFVIII (Advate®) prophylaxis from January 2014 to May of 2016. Bayesian model (myPKFit®) was employed to perform an individual PK profile using the retrospective data of rFVIII levels. PK parameters analyzed were: clearance (Cl); steady state volume (Vss); plasma half-life (t1/2); and time to reach rFVIII levels <1% (T1%). Intraindividual and interindividual coefficients of variation (CV) of the t1/2 were calculated. Besides, Kruskal-Wallis test (R® version 3.1.2) was employed in comparisons between t1/2 and clinical variables: annualized bleeding rate (ABR), annualized joint bleeding rate (AJBR), Gilbert score (GS), Pettersson scale (PS) & lower extremity affected joints detected by NMR. Results Nineteen patients were analyzed, with a mean age of 32 years (SD: 11.3; range 11-46) and 86 PK monitoring (4.5 per patient). Two patients with <15 years were excluded because t1/2 was lower than adult patients. The mean PK values in adult patients were: Cl 2.9 (0.40) mL/h/kg; Vss 50.0 (<0.001) ml/kg; t1/2 14.1 (2.1) h; and T1% 74.4 (14.4) h. The mean intraindividual CV in t1/2 was 3.6% (SD 0.02; range 0.3-6.6), whereas interindividual CV was 14.8%. We categorized t1/2 in short (<p25: 12.3 h), normal (p25-p75) and long (>p75 14.4 h), with average age of 19.5; 39.4 & 35.8 years, respectively. We detected significant differences between t1/2 and median values of joint state, but not in ABR and AJBR. The mean values of joint scores were: PS (5.5, 22.1; 17; p=0.028), GS (1, 25.9, 17.6; p= 0,008) and NMR (1.2, 2.6, 2.0; p=0.042) for short, normal and long t1/2, respectively. The limited number of patients only allows observe significant differences in patients with short t1/2, patients that also have significantly lower age (p=0.007). The younger age of these patients also justifies the lower joint damage observed. After excluding two patients <15 years significant differences in the scores of the joint state disappeared, showing that age could be a confounding variable. Conclusion PK monitoring showed a low intraindividual variability in t1/2, but significant interindividual CV. Age could modify PK parameters, so it should be assessed in an integrated manner with other clinical variables. Bayesian estimate with MyPKFit® allows know the PK profile of each patient and could be a useful tool to individualize prophylaxis adjusting by the physical activity and the bleeding pattern. We are performing a personalized one-year program to identify and treat the specific causes of poor bleeding control in prophylaxis therapy, and these are our preliminary results. Acknowledgments: This study will be supported by Baxalta grant "H15-29403". Disclosures Cid: Baxalta Innovations GmbH, now part of Shire: Other: Investigator Clinical Studies.


Author(s):  
Khudair Al-bedri ◽  
Zainab A Mahmood ◽  
Aqeel Hussein Sadeq

Objective: Psoriatic arthritis (PsA) is a chronic inflammatory arthropathy commonly associated with psoriasis, a large number of patients with PsA that has persistent inflammation may develop progressive joint damage and disability and have reduced life expectancy. This study aims to determine the associations of anti-cyclic citrullinated peptide (CCP) antibodies in adult patients with psoriasis and PsA.Methods: A case–control study was conducted at Baghdad teaching hospital, Units of Rheumatology and Dermatology, during the period from December 2016 to April 2017 including three groups; 40 patients with psoriasis, 40 patients with PsA fulfilling the CASPAR classification criteria, and 40 healthy control persons.Results: Two patients with psoriasis (5%) and 10 patients with PsA (25%) were seropositive for anti-CCP antibodies (ACPA). Healthy controls were negative for anti-CCPA. Regarding ACPA positivity, a significant difference was found between those three studied groups (p<0.001) and there was a significant difference between positive and negative ACPA in PsA patients regarding disease activity score (p=0.044).Conclusion: Positive ACPA were found more significant with PsA than in patients with psoriasis as well as associated with higher disease activity.


2017 ◽  
Vol 13 (1-2) ◽  
pp. 12-20
Author(s):  
M.B. Dzhus ◽  
H.V. Mostbauer ◽  
T.A. Karasevska ◽  
O.I. Ivashkivsky

Relevance. The existence of associations between histocompatibility antigens and JIA variants has been proved. There is no consensus that the JIAs associated with HLA-B27 antigen are transformed in adulthood into other diseases for which it is necessary to revise the diagnosis, according to the adult classification of rheumatic diseases. Is this one process that began in childhood and continues into adulthood, and whether these two processes that begin in childhood and adulthood have common signs and differences? There is few data about the hallmarks of the disease and outcome in adulthood.   Objective. – To investigate the frequency of HLA-B27 detection in adult patients with a history of JIA and to evaluate the clinical features of the course of arthritis in adulthood and the long-term articular and extra-articular consequences of JIA. Materials and methods. A survey of 132 young adult patients with different variants of JIA in the history (70 women, 62 males), aged – 24,3±8,3 years; disease duration – 13,6±9,3 years. We evaluated body mass index, anamnestic data, visual analogue scale, C-reactive protein quantitatively, HLA-B27, rheumatoid factor (RF), IgG-antibody to cyclic citrulline peptide (anti-CCP) and antinuclear antibody (АNА). Long-term effects JIA assessed by joint indices (JADI-A) and extraarticular (JADI-E) damage. Disease activity in childhood and adulthood was assessed on a scale JADAS (Juvenile Arthritis Disease Activity Score) and DAS 28. For statistic analises we use the Statistica 6.0 software packages Copyright © StatSoft, Inc. 1984-2001. Results. HLA-B27 was found in 38 patients with JIA (28,8 %), including 95 % of patients with enthesitis-related arthritis, 28,1 % – with persistent oligooarthritis and 36,8 % of patients with extended oligoarthritis, 8,3 % – with RF-positive JIA and 10,5 % – with the systemic onset JIA. According adult classification 61,5 % of patients with positive HLA-B27 antigen in adulthood developed ankylosing spondilitis or undifferentiated spondiloarthritis, in 22,7 % – undifferentiated arthritis, 100 % – psoriatic arthritis and 31,8 % – remission of the disease. In the childhood in HLA-B27 (+) patients, symmetrical joint damage (20,5 %, p<0,0001), enthesitis (20,5 %, p<0,05), lesion of the joints of the hands (26,4 %, p<0,05), defeat of more than 3 peripheral joints (36,8 %, p <0,05) and longer morning stiffness (Ме 40 [20; 60] min, p<0,001) were observed more often, compared with adult age. In adulthood, pain in the spine was significantly more frequent (27,5 %, p<0,01), as well as sacroilitis (15,0 %, p<0,05) and oligoarthritis (45 %, p<0,01). Only 21 % HLA-B27-positive patients received NSAIDs, 26,3 % had one DMARD and NSAIDs, 31,6 % had more than one DMARDs, and 21,1 % had a combination of different DMARDs and biological therapy (BTs). In childhood 58,3 % of patients received glucocorticoids and in adulthood only 22,2 % of patients but this difference was not significant. 42,1 % of adults needed intensification of therapy, 26,3 % of patients required BT. The most significant joint damages (JADI-A) in adulthood were found in the anti-CCP/RF-positive patients (3,04±4,90), whereas HLA-B27- positive patients had the lowest rates of this index, that shows the development of less remote negative consequences. Extra-articular damages (JADI-E) were most pronounced in АNА-positive patients (1,31±1,49), compared with a more favorable course in the groups anti-CCP/RF- (0,38±0,70; p <0,05) and HLA-B27-positive (0,50±1,06; p <0,05) patients. Conclusion. Clinical manifestations of articular syndrome have certain age-related pecularities in HLA-B27-positive patients with JIA: symmetrical joint damage, enthesitis, lesion of the joints of the hands, affections of more than 3 peripheral joints, and more prolonged morning stiffness are observed in childhood but in adulthood, pain in the spine, sacroilitis and oligoarticular lesion are more common. The presence of HLA-B27 antigen in patients with JIA is associated with the development of a smaller number of long-range articular damage (JADI-A), compared to the anti-CCP/RF-positive group (p <0,05) and less of the remote extra-articular effects (JADI- E) compared with the group of ANA-positive patients (p <0,05) in an adulthood.


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