scholarly journals FRI0487 APREMILAST IN MONOTHERAPY OR COMBINED IN NON-ULCER MANIFESTATIONS OF BEHÇET’S DISEASE. NATIONAL MULTICENTER STUDY OF 34 REFRACTORY CASES OF CLINICAL PRACTICE

2020 ◽  
Vol 79 (Suppl 1) ◽  
pp. 841.1-842
Author(s):  
A. Herrero Morant ◽  
B. Atienza Mateo ◽  
J. Loricera ◽  
V. Calvo del Rio ◽  
J. L. Martín-Varillas ◽  
...  
Keyword(s):  
Standard Dose ◽  
Clinical Manifestations ◽  
Skin Lesions ◽  
Research Support ◽  
Open Label ◽  
Biologic Dmards ◽  
Aphthous Ulcers ◽  
Musculoskeletal Manifestations ◽  
Del Rio

Background:Apremilast (APR) has demonstrated efficacy in orogenital ulcers of Behçet´s disease (BD). Response of other clinical manifestations remains unknown.Objectives:To assess the efficacy and safety of APR in monotherapy or combined with disease-modifying anti-rheumatic drugs (DMARDs) in non-aphthous ulcers of BD.Methods:National multicenter open-label study on 34 BD patients treated with APR at maintained standard dose of 30 mg twice daily.Results:From a cohort of 51 patients with APR by refractory orogenital ulcers of BD, we selected 34 (24 women/10 men, mean age 43.8±14.3 years), cases with another clinical manifestation/s.Excluding CTs, colchicine or NSAIDs, APR was given in monotherapy (n=21) or combined with conventional and/or biologic DMARDs in 13 cases (5 methotrexate, 3 azathioprine, 3 hydroxychloroquine, 1 sulfasalazine, 1 dapsone, 2 tocilizumab, 1 IFX). Other active manifestations present at APR onset were: arthralgia/arthritis (16, true arthritis in 5), folliculitis/pseudofolliculitis (14), erythema nodosum (3), furunculosis (2), paradoxical psoriasis by TNFi (2), intestinal ileitis (2), deep venous thrombosis (2), leg ulcers (1), erythematosus and scaly skin lesions (1), fever (1), unilateral anterior uveitis (1) and neurobehçet (1).After a median follow-up of 6 [3-12] months, folliculitis and ileitis improved, neurobehçet remained stable and musculoskeletal manifestations evolved in a variable way.(TABLE)TABLE.Conclusion:In addition of orogenital ulcers, APR in monotherapy or combined, seems to be useful in skin manifestations of BDDisclosure of Interests:Alba Herrero Morant: None declared, Belen Atienza Mateo: None declared, J. Loricera: None declared, Vanesa Calvo del Rio Grant/research support from: MSD and Roche, Speakers bureau: Abbott, Lilly, Celgene, Grünenthal, UCB Pharma, José Luis Martín-Varillas Grant/research support from: AbbVie, Pfizer, Janssen and Celgene, Speakers bureau: Pfizer and Lilly, Gerard Espinosa: None declared, Jenaro Graña: None declared, Clara Moriano: None declared, Trinidad Pérez Sandoval: None declared, Manuel Martín Martínez: None declared, Elvira Diez: None declared, María Dolores García-Armario: None declared, Esperanza Martínez: None declared, Ivan Castellví Consultant of: Boehringer Ingelheim, Actelion, Kern Pharma, Speakers bureau: Boehringer Ingelheim, Actelion, Bristol-Myers Squibb, Roche, Patricia Moya Alvarado: None declared, Francisca Sivera: None declared, Jaime Calvo Grant/research support from: Lilly, UCB, Consultant of: Abbvie, Jansen, Celgene, Isabel de la Morena: None declared, Francisco Ortiz Sanjuán: None declared, José Andrés Román Ivorra: None declared, Ana Pérez Gómez: None declared, Alejandro Olive: None declared, Carolina Díez: None declared, Juan José Alegre: None declared, D Ybáñez-García Speakers bureau: Lilly, Roche, Sanofi, Ángels Martínez-Ferrer: None declared, Javier Narvaez: None declared, Ignasi Figueras: None declared, Ana Isabel Turrión: None declared, Susana Romero-Yuste: None declared, Pilar Trénor: None declared, Soledad Ojeda Speakers bureau: AMGEN, LILLY, GEBRO, Miguel Á. González-Gay Grant/research support from: AbbVie, MSD and Roche, Speakers bureau: AbbVie, MSD and Roche, Ricardo Blanco Grant/research support from: Abbvie, MSD and Roche, Consultant of: Abbvie, Pfizer, Roche, Bristol-Myers, Janssen and MSD, Speakers bureau: Abbvie, Pfizer, Roche, Bristol-Myers, Janssen, Lilly and MSD

scholarly journals Apremilast in refractory orogenital ulcers and other manifestations of Behçet's disease. National multicenter study of 51 cases in clinical practice.

2020 ◽  
Author(s):  
Belén Atienza-Mateo ◽  
José Luis Martín-Varillas ◽  
Jenaro Graña ◽  
Gerard Espinosa ◽  
Clara Moriano ◽  
...  
Keyword(s):  
Standard Dose ◽  
Clinical Manifestations ◽  
Skin Lesions ◽  
Open Label ◽  
Biologic Drugs ◽  
Phosphodiesterase 4 ◽  
Aphthous Ulcers ◽  
Tnfα Inhibitors ◽  
Musculoskeletal Manifestations ◽  
Genital Ulcers

Abstract Background Oral and/or genital aphthous ulcers are the most common symptoms of Behçet´s disease (BD), and are often refractory to conventional treatment. The inhibitor of phosphodiesterase-4 apremilast (APR) has demonstrated efficacy in the treatment of this manifestations. The objective of the present study was to assess the efficacy of APR in the management of refractory oral and/or genital ulcers in patients with BD. Methods National multicenter open-label observational study on BD patients with recurrent oral and/or genital ulcers. In all cases orogenital ulcers were refractory to conventional therapy. APR was given and maintained at standard dose of 30 mg twice daily. The main outcome was the achievement of oral and/or genital ulcers remission. Efficacy of APR for other clinical manifestations was also evaluated. Results We included 51 patients (35 women/16 men; mean age 44.7 ± 13.2 years). Before APR, all patients had received several systemic conventional and/or biologic drugs. APR was initiated because of refractory oral (n = 19) or genital (n = 2) aphthous ulcers or both (n = 30). Other manifestations found at APR onset were arthralgia/arthritis (n = 16), folliculitis/pseudofolliculitis (n = 14), erythema nodosum (n = 3), furunculosis (n = 2), paradoxical psoriasis induced by TNFα-inhibitors (n = 2), ileitis (n = 2), deep venous thrombosis (n = 2), leg ulcers (n = 1), erythematosus and scaly skin lesions (n = 1), fever (n = 1), unilateral anterior uveitis (n = 1) and neurobehçet (n = 1). After a mean follow-up of 8.5 ± 6.9 months, most patients had experienced improvement of orogenital ulcers and prednisone dose had been successfully reduced or discontinued. APR also yielded improvement of some non-aphthous manifestations such as the cutaneous follicular and intestinal manifestations. However, the effect on musculoskeletal manifestations was variable. Conclusion APR yielded a rapid and maintained improvement of refractory mucocutaneous ulcers of BD, even in patients refractory to several systemic drugs including biologic therapy.

scholarly journals POS0296 DOSING OF BDMARDS IN AXSPA AND PSA IN A REAL WORLD SETTING

2021 ◽  
Vol 80 (Suppl 1) ◽  
pp. 373.1-373
Author(s):  
A. Regierer ◽  
A. Weiß ◽  
D. Poddubnyy ◽  
H. Kellner ◽  
F. Behrens ◽  
...  
Keyword(s):  
Real World ◽  
Standard Dose ◽  
Research Support ◽  
Biologic Dmards ◽  
Real World Setting ◽  
The Mean ◽  
Receive Treatment ◽  
Prospective Longitudinal ◽  
Higher Doses

Background:The treatment of patients with axial spondyloarthritis (axSpA) and psoriatic arthritis (PsA) has been revolutionised by the introduction of biologic DMARDs targeting TNF, IL17, and IL23 inhibitors (i). In Germany, about 30-50% of axSpA and PsA patients receive treatment with bDMARDs. Although many patients benefit from these drugs, in some patients effectiveness of the standard dose may be insufficient and higher doses are used.Objectives:To describe dosing of TNFi and non-TNFi bDMARDs over a 2 year period in a real world cohort of patients with axSpA and PsA managed by rheumatologists.Methods:RABBIT-SpA is a prospective longitudinal cohort study including axSpA and PsA patients enrolled at the start of a new conventional treatment (including NSAID) or b/tsDMARD treatment. Description of dosing of TNFi (adalimumab bio-original (bo), adalimumab bio-similar (bs), etanercept bo, etanercept bs, golimumab, certolizumab) in comparison to nonTNFi-bDMARDs (secukinumab, ustekinumab, ixekizumab, guselkumab) in axSpA and PsA. Standard dosing was defined according to the current labels for axSpA and PsA.Results:1628 patients (axSpA: n=903, PsA: n=725) were included in this analysis. At inclusion mean age was 44 years in axSpA and 51 years in PsA. 44% of patients with axSpA and 58% of those with PsA were female. The mean disease duration of axSpA was 7.6 years, of PsA 6.4 years.Standard doses of TNFi were used during a 2 year period in > 90% of patients with axSpA and PsA (Figure 1). In contrast, standard doses of non-TNFi-bDMARDs were only used in 70-80% of patients. The percentage of documented higher doses in patients with axSpA ranged from 20-30% at different time points. In PsA, this percentage increased from 27% at baseline to 44% at 2 years. On the other hand, TNFi were used in lower doses than the label in up to 9% and 7 % of patients with axSpA and PsA, respectively, after 2 years.Figure 1.Percentages of patients with axSpA or PsA who received less than, equal to, or more than the approved doses of bDMARDs at baseline and at 5 follow-up visits.Conclusion:While TNFi are used in licensed doses in most patients, non-TNFi-bDMARDs were often used in higher doses, which corresponds to higher doses approved in other indications like psoriasis. The effectiveness of this treatment strategy in axSpA and PsA needs to be analysed further.Acknowledgements:RABBIT-SpA is supported by a joint, unconditional grant from AbbVie, Amgen, Biogen, Hexal, Janssen-Cilag, Lilly, MSD, Novartis, Pfizer, UCB, and Viatris.We thank all participating patients and rheumatologists.Disclosure of Interests:Anne Regierer Grant/research support from: AbbVie, Amgen, Biogen, Hexal, Janssen-Cilag, Lilly, MSD, Novartis, Pfizer, UCB, and Viatris., Anja Weiß Grant/research support from: AbbVie, Amgen, Biogen, Hexal, Janssen-Cilag, Lilly, MSD, Novartis, Pfizer, UCB, and Viatris., Denis Poddubnyy: None declared, Herbert Kellner: None declared, Frank Behrens: None declared, Georg Schett: None declared, Juergen Braun: None declared, Joachim Sieper: None declared, Anja Strangfeld Grant/research support from: AbbVie, Amgen, Biogen, Hexal, Janssen-Cilag, Lilly, MSD, Novartis, Pfizer, UCB, and Viatris

Follow-up results of myositis patients treated with H. P. Acthar gel

Rheumatology ◽  
2020 ◽  
Vol 59 (10) ◽  
pp. 2976-2981 ◽  
Author(s):  
Didem Saygin ◽  
Chester V Oddis ◽  
Galina Marder ◽  
Siamak Moghadam-Kia ◽  
Preeya Nandkumar ◽  
...  
Keyword(s):  
Standard Dose ◽  
Manual Muscle Testing ◽  
Open Label ◽  
Muscle Testing ◽  
Core Set ◽  
Minimal Improvement ◽  
Definition Of ◽  
Post Trial ◽  
Lost To Follow Up

Abstract Objectives Idiopathic inflammatory myopathies (IIM) are a group of autoimmune diseases characterized by proximal muscle weakness. H. P. Acthar gel [repository corticotropin injection (RCI)] is a formulation of adrenocorticotropic hormone and has been approved by Food and Drug Administration for use in IIM; however, literature is limited. In this study, we report longitudinal follow-up of myositis patients treated with RCI. Methods Patients with refractory IIM who were enrolled in the prospective, open-label RCI trial were included in this study. The post-trial follow-up period was 6 months with assessments every 2 months, which included myositis core set measures including extra-muscular global, muscle and patient global disease activities, HAQ, and manual muscle testing. Results Two patients were lost to follow-up after finalization of the trial, and the remaining eight patients were enrolled in the follow-up study. One patient remained on RCI after the trial. In the follow-up period, four of eight patients had flare at on average 4.1 months after the RCI trial. Among the patients who flared, three required an increase in prednisone. One patient was restarted on RCI at 5.5 months, but had minimal improvement after 3 months. Four patients who remained stable continued to satisfy criteria for the definition of improvement through the 6-month follow-up. However, none showed any further improvement in the primary or secondary efficacy outcomes after the initial RCI trial. Conclusion To our knowledge, this is the first study reporting the follow-up results of patients treated with standard dose and duration of Acthar. We believe that our study will provide the basis for the development of future randomized RCI trials in IIM.

scholarly journals Essential syphilitic alopecia: Non-scarring alopecia in 21-year-old man

2021 ◽  
Vol 12 (e) ◽  
pp. 1-3
Author(s):  
Hafssa Chehab ◽  
Bertrand Richert
Keyword(s):  
Hair Loss ◽  
Previous History ◽  
Treponema Pallidum ◽  
Clinical Manifestations ◽  
Skin Lesions ◽  
Secondary Syphilis ◽  
Laboratory Evaluation ◽  
History Of ◽  
High Level

ABSTRACT Alopecia syphilitica is a less common clinical manifestations of secondary syphilis. It is uncommon for hair loss to be the sole or predominant manifestation, as hair loss is the chief clinical and histologic differential diagnosis of. The main difference between alopecia areata and Alopecia syphilitica is the detection of Treponema pallidum in syphilis. We present the case of a 21- year-old belgium man with different patches of non-cicatricial alopecia of his scalp. The patient denied previous history of genital or other skin lesions. Laboratory evaluation was positive for syphilis. The diagnosis of alopecia syphilitica was made and he was treated with single intramuscular injections of benzathine penicillin. The lesions improved with treatment in all the patients who attended follow-up. Dermatologists should maintain a high level of clinical suspicion for this uncommon manifestation of syphilis, particularly when it is the only symptom.

scholarly journals AB0373 TREATMENT OF SLE WITH THE IMMUNOPROTEASOME INHIBITOR KZR-616: RESULTS FROM THE FIRST 4 COHORTS OF THE MISSION STUDY, AN OPEN-LABEL PHASE 1B DOSE ESCALATION TRIAL

2020 ◽  
Vol 79 (Suppl 1) ◽  
pp. 1486.1-1487
Author(s):  
R. Furie ◽  
S. Parikh ◽  
A. Maiquez ◽  
A. Khan ◽  
O. Moreno ◽  
...  
Keyword(s):  
Peripheral Neuropathy ◽  
Disease Activity ◽  
Dose Escalation ◽  
Global Assessment ◽  
Life Sciences ◽  
Research Support ◽  
Open Label ◽  
Long Term Treatment ◽  
Phase 1B

Background:Non-specific proteasome inhibitors, such as bortezomib (BTZ), target the constitutive proteasome and immunoproteasome and are approved treatments for multiple myeloma1. BTZ has also been used to treat systemic lupus erythematosus (SLE) and lupus nephritis (LN); however, treatment emergent adverse events (TEAEs), such as gastrointestinal (GI) effects, hematologic abnormalities, asthenia and peripheral neuropathy, limit its use as a long-term treatment option for chronic autoimmune disease2. KZR-616 is a first-in-class selective immunoproteasome inhibitor and is highly active in murine SLE3. Subcutaneous (SC) administration of KZR-616 (30 and 45 mg weekly [QW]) was demonstrated as safe and well-tolerated, and successfully achieved target levels of immunoproteasome inhibition in healthy volunteers4, 5.Objectives:We report the preliminary safety and efficacy of KZR-616 in the first 4 cohorts of the Phase 1b portion of Study KZR-616-002 in patients with active SLE (NCT03393013).Methods:SLE patients (per SLICC Classification Criteria) with SLEDAI ≥4 despite stable background immunosuppressant, anti-malarial, and/or corticosteroid (≤20 mg prednisone equivalent) therapy in this open-label multicentric dose-escalation trial received KZR-616 at doses of 45mg (Cohort 1), 60mg (Cohort 2), or 30mg with escalation to 60mg (Cohorts 2a and 2b) SC weekly through Week 13 (W13) with 12 weeks of follow-up. Efficacy measures included SLEDAI, Cutaneous Lupus Erythematous Disease Area and Severity Index, 28 tender and swollen joint counts, Physicians Global Assessment, Patient Global Assessment, and Patient Assessment of Pain, in evaluable patients (those who received ≥1 month of KZR-616).Results:As of 16 January 2020, 33 patients had enrolled and received at least 1 dose of KZR-616. The majority of TEAEs have been mild or moderate with no reported peripheral neuropathy, prolonged GI-related AEs, and no clinically significant laboratory AEs. There were 3 treatment emergent SAEs, one each of thrombotic microangiopathy (Cohort 2), localized herpes zoster (Cohort 2a), and systemic inflammatory response syndrome (Cohort 2a) with the latter 2 patients completing the full 13 weeks of treatment after resolution. When compared to baseline, improvement in all measures of disease activity were seen at W13 and maintained or improved during the follow-up period, and 94% of evaluable patients had improvements on at least 2 measures/assessments of disease activity. A single patient with active class IV/V nephritis was enrolled on prednisone 10 mg, leflunomide 10 mg, and hydroxychloroquine 200mg/day; nephrotic-range proteinuria at baseline (3.85 g/day) decreased to 0.6 g/day 4 weeks after the last dose of KZR-616.Conclusion:Weekly subcutaneous administration of KZR-616 at 45 and 60 mg was safe and well-tolerated. Evidence of disease suppression at W13 in active SLE patients on stable background therapy was observed. In addition, one study participant with active proliferative nephritis was enrolled with significant reduction in proteinuria. The Phase 2 portion of this study in active proliferative LN is open for enrollment.References:[1]Scott K.et al.Cochrane Database Syst Rev 2016;4:CD010816[2]Alexander Tet al.Ann Rheum Dis 2015;74: 1474–8[3]Muchamuel T.et al.Ann Rheum Dis 2018;77: A685[4]Lickliter J.et al.Ann Rheum Dis 2018;77: A1413[5]Furie Ret al.Ann Rheum Dis 2019;78: A776Disclosure of Interests:Richard Furie Grant/research support from: AstraZeneca, Biogen, Consultant of: AstraZeneca, Biogen, SV Parikh Grant/research support from: Aurinia Pharmaceuticals, EMD-Serono, Consultant of: Aurinia, BMS, GSK, Adonis Maiquez: None declared, Amber Khan: None declared, Orlando Moreno: None declared, Miguel Soneira: None declared, Christopher Kirk Shareholder of: Kezar Life Sciences, LLC, Employee of: Kezar Life Sciences, LLC, Darrin Bomba Shareholder of: Kezar Life Sciences, LLC, Employee of: Kezar Life Sciences, LLC, Ken Harvey Shareholder of: Kezar Life Sciences, LLC, Employee of: Kezar Life Sciences, LLC, Mary Katherine Farmer Shareholder of: Kezar Life Sciences, Employee of: Kezar Life Sciences

AB0752 PSORIATIC ARTHRITIS: OLIGOARTHRITIS AND POLYARTHRITIS PATTERN CHANGES OVER THE INITIAL YEAR OF THE PRESENTATION. A REAL-WORLD EVIDENCE REPORT FROM THE QUEBEC REGISTRY RHUMADATA®

2020 ◽  
Vol 79 (Suppl 1) ◽  
pp. 1672.1-1673
Author(s):  
S. Chambah ◽  
L. Coupal ◽  
D. Choquette
Keyword(s):  
Psoriatic Arthritis ◽  
Clinical Manifestations ◽  
Original Description ◽  
Joint Disease ◽  
First Year ◽  
Research Support ◽  
Real World Evidence ◽  
The Mean ◽  
Relationship Of

Background:Psoriatic Arthritis (PsA) most frequently presents as a polyarthritis or (less often) as an oligoarthritis [1]. Upon reassessment, patients may change category during follow-up [2-3]. Historically, the patients in the original description of Moll and Wight had an oligoarticular presentation [4]. However, other studies have not found the same distribution in all patient populations [5]. Currently, none of the accepted diagnostic or classification criteria set for PsA consider the variation in the number of involved joints in the early phase of PsA.Objectives:To evaluate the change in pattern between oligoarticular and polyarticular psoriatic arthritis, within the first year of follow-up.Methods:Data from RHUMADATA® patients diagnosed with PsA were extracted on December 8th, 2019. In the current analysis, we consider the first year of care patients following their first encounter with clinic staff. Patients with at least two 66/68 joint counts completed during this initial year are the subjects of this analysis. Joint count classification (Oligo vs Poly) was assessed from the first and last available joint counts. Patients were classified as having a polyarticular form of PsA if 5 or more of their joints were assessed as being swollen and/or tender. Subjects with 4 or less swollen and/or tender joints were classified as oligoarticular PsA patients.Results:A total of 287 patients with at least two 66/68 joint counts are used in the present analysis. At baseline, the mean age of patients was 47.8 ± 13.5 with average disease duration of 1.6 ± 5.2 years. 49 % of patients were women. Average joint count at baseline was 7.1 ± 7.2 (swollen) and 7.1 ± 7.5 (tender) joints. Considering only 28 joints, the average was 4.2 ± 5 and 3.9 ± 4.8 for swollen and tender joints respectively. At the first joint count, 115 (40%) patients were assessed as “Oligo” and 172 (60%) as “Poly”, while 159 (55%) and 128 (45%) were similarly assessed at the last assessment. The two assessments agreed for 179 (62%) and disagreed for 108 (38%). Of the 115 patients initially classified as “Oligo”, 32 (28%) were reassessed as “Poly” within the initial year, while 76 (44%) of the 172 patients initially classified as “Poly” were reassessed as “Oligo”. All 172 patients initially classified as “Poly” initiated a DMARD during this period (167 (97%) initiated a csDMARD and 5 (3%) initiated a bDMARS). All patients initially classified as “Oligo” also initiated treatment during this period (98 (85%) and 17 (15%) of the 115 patients initially classified as “Oligo” initiated csDMARDs and bDMARD respectively).Conclusion:These observations suggest that a single assessment of joint count may be misleading in establishing the oligo or polyarticular pattern of PsA. This classification should take treatment into account.References:[1]Gladman DD, Ritchlin C, et al. Clinical manifestations and diagnosis of psoriatic arthritis. Uptodate 2019.[2]Jones SM, Armas JB, Cohen MG, et al. Psoriatic arthritis: outcome of disease subsets and relationship of joint disease to nail and skin disease. Br J Rheumatol 1994; 33:834.[3]McHugh NJ, Balachrishnan C, Jones SM. Progression of peripheral joint disease in psoriatic arthritis: a 5-yr prospective study. Rheumatology (Oxford) 2003; 42:778.[4]Wright V, Moll JM. Psoriatic arthritis. Bull Rheum Dis 1971; 21:627.[5]Gladman DD. Psoriatic arthritis. Baillieres Clin Rheumatol 1995; 9:319.Disclosure of Interests:Sana Chambah: None declared, Louis Coupal: None declared, Denis Choquette Grant/research support from: Rhumadata is supported by grants from Pfizer, Amgen, Abbvie, Gylead, BMS, Novartis, Sandoz, eli Lilly,, Consultant of: Pfizer, Amgen, Abbvie, Gylead, BMS, Novartis, Sandoz, eli Lilly,, Speakers bureau: Pfizer, Amgen, Abbvie, Gylead, BMS, Novartis, Sandoz, eli Lilly,

scholarly journals Congenital syphilis in Argentina: experience in a pediatric hospital

2020 ◽  
Author(s):  
Luciana Noemí Garcia ◽  
Alejandra Destito Solján ◽  
Nicolas Falk ◽  
Nicolás Leonel Gonzalez ◽  
Griselda Ballering ◽  
...  
Keyword(s):  
Medical Records ◽  
Congenital Syphilis ◽  
Delayed Diagnosis ◽  
Clinical Manifestations ◽  
Skin Lesions ◽  
Penicillin G ◽  
High Morbidity ◽  
Laboratory Findings ◽  
Therapeutic Decisions

AbstractAlthough congenital syphilis (CS) is preventable, it is still an important health problem worldwide. Recently, an increase in the number of primary and congenital syphilis cases has been observed. Fetal infection can be particularly aggressive, but newborns can be asymptomatic at birth and run the risk of developing systemic compromise with a poor prognosis.We conducted a study (1987-2019) analyzing the medical records of CS diagnosis cases assisted at the Buenos Aires Children’ Hospital. Sixty-one patients were included. Information about demographics, clinical and laboratory findings, T. pallidum serology and treatment was collected. Median age at diagnosis was 2 months (IQ 1-6 months). The distribution of cases showed a bimodal curve, with a peak in 1993 and in 2017. The main clinical findings were: bone alterations in 36/61 (59%); hepatosplenomegaly in 33/61 (54.1%); anemia in 32/51 (62.8%); skin lesions 26/61 (42.6%) and renal compromise in 15/45 (33.3%). Cerebrospinal fluid was studied in 50/61 (81.9%); 5 (10%) were abnormal (reactive VDRL and/or cell alteration count). Only 23 (60.5%) patients had nontreponemal titers fourfold higher than their mothers did. Intravenous penicillin G for an average of 10-14 days was prescribed in 60/61 subjects and one patient received ceftriaxone. Remarkably, only 28 (46%) mothers were tested for syphilis during pregnancy.During follow-up, a decrease in RPR titers was observed reaching seroconversion in 31/34 (91%) subjects at a median of 19.2 months after treatment. Treponemal titers (TPHA) remained reactive.Our results highlighted that an increase in the number of cases of CS is occurring in our population with high morbidity related to delayed diagnosis. A good therapeutic response was observed.CS requires a greater effort from obstetricians to adequately screen for the disease during pregnancy and pediatricians should be alert in order to detect cases earlier, to provide an adequate diagnosis and treatment of CS.Author SummaryCS is caused by mother-to child transmission. Although screening of pregnant women and treatments are available, new cases are increasing worldwide. We reviewed the medical records of CS-patients assisted in our hospital over the past 30 years. Our results showed that there was an increase in the number of CS cases. At birth, most children were asymptomatic and later developed CS clinical manifestations. Penicillin treatment, and in one case ceftriaxone, was prescribed with a good clinical response. Nevertheless, one infant died, four had persistent kidney disorders and one showed bone sequelae damage. Spinal lumbar puncture did not modify therapeutic decisions. In the follow-up, a decrease in nontreponemal antibodies was observed as a marker of treatment response. We concluded that the detection and treatment of CS remains a great challenge for clinical practice in our region.It is crucial that pediatricians and obstetricians give greater attention and make a greater effort to detect this neglected disease in an attempt to reverse its upwards trend.

scholarly journals OP0209 INTERVAL PROLONGATION IN ETANERCEPT-TREATED PATIENTS WITH RHEUMATOID ARTHRITIS, ANKYLOSING SPONDYLITIS OR PSORIATIC ARTHRITIS: AN OPEN-LABEL, RANDOMISED CONTROLLED TRIAL

2020 ◽  
Vol 79 (Suppl 1) ◽  
pp. 130-130
Author(s):  
M. J. L’ami ◽  
J. Ruwaard ◽  
E. L. Kneepkens ◽  
C. L. M. Krieckaert ◽  
M. Nurmohamed ◽  
...  
Keyword(s):  
Rheumatoid Arthritis ◽  
Standard Dose ◽  
Controlled Trial ◽  
Intervention Group ◽  
Control Group ◽  
Interval Prolongation ◽  
Open Label ◽  
Dosing Interval ◽  
Randomised Controlled

Background:The majority of patients with a rheumatic disease treated with etanercept may be overexposed. Data regarding etanercept tapering is scarce, particularly in psoriatic arthritis (PsA) and ankylosing spondylitis (AS). Dose reductions can potentially reduce blood drug levels too much, resulting in loss of effect.Objectives:We compared extending the dose interval to continuation of the standard dose and studied the success rate of etanercept discontinuation. Etanercept concentrations were measured throughout the study.Methods:160 consecutive patients with rheumatoid arthritis (RA), PsA or AS with sustained minimal disease activity (MDA) were enrolled in this 18-month, open-label, randomised controlled trial. The intervention group doubled the dosing-interval at baseline and discontinued etanercept 6 months later. The control group continued the standard dose up to 6 months, after which the dosing-interval was doubled. Primary outcome was the proportion of patients maintaining MDA after 6 months follow-up.Results:At 6 months, MDA status was maintained in 47 (63%) patients in the intervention group and 56 (74%) in the control group (p=0.15), with comparable results in all rheumatic diseases. Median etanercept concentrations decreased from 1.50 µg/mL (25-75thpercentile 1.06-2.65) to 0.46 µg/mL (0.28-0.92) after 6 months of interval prolongation (figure 1). In total, 40% discontinued etanercept successfully with maintained MDA for at least 6 months.Figure 1.Median (with Q1 to Q3 boxplots) etanercept concentrations (per protocol) during the first 6 months of follow-up in the intervention group (prolongation; gray boxplots) and the control group (continuation; white boxplots), separated by disease (RA, PsA, AS). Bars represent 10-90 percentile and outliers are shown separately (dots).Conclusion:As observed in RA, etanercept tapering can be safely attempted in PsA and AS patients in sustained MDA. A substantial proportion of patients could stop etanercept for at least 6 months. In many patients low drug concentrations proved sufficient to control disease activity. However, the risk of minor and major flares is substantial, even in patients continuing standard dosing.References:noneDisclosure of Interests:Merel J. l’Ami Speakers bureau: Novartis, Jill Ruwaard: None declared, Eva L. Kneepkens: None declared, Charlotte L.M. Krieckaert: None declared, Michael Nurmohamed Grant/research support from: Not related to this research, Consultant of: Not related to this research, Speakers bureau: Not related to this research, Femke Hooijberg: None declared, J.C. van Denderen: None declared, Arno Van Kuijk: None declared, Lot Burgemeister: None declared, Maarten Boers: None declared, Gert-Jan Wolbink: None declared

scholarly journals OP0228 EFFICACY AND SAFETY OF IXEKIZUMAB VERSUS ADALIMUMAB (SPIRIT-H2H) WITH AND WITHOUT CONCOMITANT CONVENTIONAL SYNTHETIC DISEASE-MODIFYING ANTIRHEUMATIC DRUGS (DMARD) IN BIOLOGIC DMARD-NAÏVE PATIENTS WITH PSORIATIC ARTHRITIS: 52-WEEK RESULTS

2020 ◽  
Vol 79 (Suppl 1) ◽  
pp. 143-144 ◽  
Author(s):  
J. S. Smolen ◽  
A. Sebba ◽  
E. Ruderman ◽  
A. Gellett ◽  
C. Sapin ◽  
...  
Keyword(s):  
Psoriatic Arthritis ◽  
Primary Endpoint ◽  
Severity Index ◽  
Current Treatment ◽  
Inadequate Response ◽  
Efficacy And Safety ◽  
Research Support ◽  
Open Label ◽  
Biologic Dmards ◽  
Logistic Regression Models

Background:Ixekizumab (IXE), a high-affinity monoclonal antibody selectively targeting IL-17A, was superior to adalimumab (ADA) at Week (Wk) 24 for simultaneous achievement of ACR50 and 100% improvement from baseline in the Psoriasis Area and Severity Index (PASI 100) (primary endpoint) in patients (pts) with active PsA from SPIRIT-H2H1. SPIRIT-H2H had two major secondary endpoints and achieved both: noninferiority of IXE to ADA for ACR50 at Wk 24, and superiority of IXE to ADA for PASI 100 at Wk 24.Objectives:To determine how concomitant conventional synthetic DMARD (csDMARD) use affects safety and efficacy of IXE and ADA in prespecified subgroups defined by biologic monotherapy, concomitant MTX use, and concomitant csDMARD use through Wk 52 in SPIRIT-H2H.Methods:SPIRIT-H2H (NCT03151551) was a 52-week, multicentre, randomised, open-label, assessor-blinded, parallel-group study evaluating the efficacy and safety of IXE versus ADA in adults with PsA and naïve to biologic DMARDs. Patients were required to have active PsA fulfilling Classification for Psoriatic Arthritis (CASPAR) criteria and ≥3/68 tender and ≥3/66 swollen joints, ≥3% plaque psoriasis BSA involvement, no prior treatment with bDMARDs, and with prior inadequate response to ≥1 csDMARD (but not necessarily current treatment with csDMARDs). Randomization (1:1) was stratified by concomitant use of csDMARD and the presence/absence of moderate to severe PsO (baseline: BSA≥10% + PASI≥12, + static Physician’s Global Assessment≥3). Patients (N=566) received IXE/ADA through 52 wks according to the labelled dose dependent on presence/absence of moderate-to-severe PsO. In this prespecified subgroup analysis by presence or absence of csDMARDs, efficacy outcomes through wk 52 were compared between IXE and ADA using logistic regression models and Fisher’s exact tests. Missing data were imputed using non-responder imputation.Results:At baseline, 167 of 283 IXE-treated patients and 169 of 283 ADA-treated patients had concomitant MTX use. Of these, 9.0% (15/167) and 7.1% (12/169) treated with IXE and ADA, respectively, were taking an additional csDMARD (sulfasalazine, cyclosporine, or leflunomide). A significantly greater proportion of patients on IXE versus ADA achieved the primary endpoint or PASI 100 when used as monotherapy or in combination with csDMARD (Figure 1A and 1C). At Wk 52, the proportion of patients achieving ACR50 was not statistically different between IXE and ADA, regardless of monotherapy or concomitant csDMARD use (Figure 1B). A significantly higher proportion of patients achieved MDA on IXE compared to ADA in the monotherapy subgroup (49% vs 33%), while the response rates were similar in both combination subgroups (Figure 1D). These data support consistent ACR50, PASI 100, and MDA response for IXE across all three subgroups. Frequencies of adverse events were similar across the three subgroups for IXE and ADA (Figure 2).Conclusion:As with prior studies,2,3consistent efficacy across multiple PsA disease-specific endpoints was observed with IXE in SPIRIT-H2H, regardless of whether IXE was taken as monotherapy or in combination with MTX or another csDMARD. No unexpected safety signals were found for either agent.References:[1]Mease et al, Ann Rheum Dis 2020;79:123-31.[2]Coates et al, RMD Open 2017;3:e000567.[3]Nash et al, RMD Open 2018;4:e000692.Disclosure of Interests:Josef S. Smolen Grant/research support from: AbbVie, AstraZeneca, Celgene, Celltrion, Chugai, Eli Lilly, Gilead, ILTOO, Janssen, Novartis-Sandoz, Pfizer Inc, Samsung, Sanofi, Consultant of: AbbVie, AstraZeneca, Celgene, Celltrion, Chugai, Eli Lilly, Gilead, ILTOO, Janssen, Novartis-Sandoz, Pfizer Inc, Samsung, Sanofi, Anthony Sebba Consultant of: Genentech, Gilead, Lilly, Regeneron Pharmaceuticals Inc., Sanofi, Speakers bureau: Lilly, Roche, Sanofi, Eric Ruderman Consultant of: Pfizer, Amanda Gellett Shareholder of: Eli Lilly and Company, Employee of: Eli Lilly and Company, Christophe Sapin Shareholder of: Eli Lilly and Company, Employee of: Eli Lilly and Company, Aubrey Trevelin Sprabery Shareholder of: Eli Lilly and Company, Employee of: Eli Lilly and Company, Soyi Liu Leage Shareholder of: Eli Lilly and Company, Employee of: Eli Lilly and Company, Sreekumar Pillai Shareholder of: Eli Lilly and Company, Employee of: Eli Lilly and Company, Paulo Reis Shareholder of: Eli Lilly and Company, Employee of: Eli Lilly and Company, Peter Nash Grant/research support from: AbbVie, Bristol-Myers Squibb, Celgene, Eli Lilly and Company, Gilead, Janssen, MSD, Novartis, Pfizer Inc, Roche, Sanofi, UCB, Consultant of: AbbVie, Bristol-Myers Squibb, Celgene, Eli Lilly, Gilead, Janssen, MSD, Novartis, Pfizer Inc, Roche, Sanofi, UCB, Speakers bureau: AbbVie, Bristol-Myers Squibb, Celgene, Eli Lilly, Gilead, Janssen, MSD, Novartis, Pfizer Inc, Roche, Sanofi, UCB

scholarly journals 595. Letermovir (LTV) for Secondary Cytomegalovirus (CMV) Prevention in High Risk Hematopoietic Cell Transplant (HCT) Recipients: Interim Results of a Single Center, Open Label Study

2021 ◽  
Vol 8 (Supplement_1) ◽  
pp. S401-S401
Author(s):  
Gyuri Han ◽  
Anat Stern ◽  
Yiqi Su ◽  
Boglarka Gyurkocza ◽  
Craig Sauter ◽  
...  
Keyword(s):  
High Risk ◽  
Research Study ◽  
Scientific Research ◽  
Cell Transplant ◽  
Research Support ◽  
Open Label ◽  
Material Support ◽  
Study Investigator ◽  
Research Grant

Abstract Background Letermovir (LTV) is effective for prevention (ppx) of primary clinically significant CMV infection (csCMVi) in the first 100 days after hematopoietic cell transplant (HCT). Data on LTV for secondary ppx is limited. We report on the efficacy and safety of LTV administered for 14 weeks as secondary CMV ppx. Methods Patients (pts) enrolled in an open label study of LTV (ClinicalTrials.gov identifier: NCT04017962) from August 2019 through February 2021 were analyzed. Key eligibility criteria were: CMV high risk (receipt of mismatched and/or T-cell depleted HCT and/or graft versus host disease (GVHD) requiring systemic immunosuppressants) AND prior csCMVi with either undetectable CMV (≤ 136 IU/mL) or ≥ 2 consecutive values < 300 IU/mL at enrollment. Pts with breakthrough csCMVi on LTV or history of LTV resistance were excluded. LTV was administered for 14 weeks or csCMVi whichever occurred first. The study duration was 24 weeks. CMV was monitored per standards of care. The primary endpoint was csCMVi by week 14. Secondary endpoints were csCMVi by week 24, LTV resistance, CMV end-organ disease (EOD) and adverse events (AE) at least possibly related to LTV. Results Of 20 pts analyzed, the median age was 58 years (interquartile range [IQR] 46-63); 17 (85%) pts were CMV seropositive, 7 (35%) received mismatched HCT (haploidentical 3, cord blood 3; mismatched unrelated 1), 9 (45%) received CD34 selected allograft and 9 (45%) had GVHD at enrollment. Fourteen (70%) pts had received prior LTV. The median time from HCT to enrollment was 156 (IQR 37-244) and 55 (IQR 40-69) days for pts with and without prior LTV, respectively (P=0.16). CMV at enrollment was < 136IU/mL for 8 (40%) pts. By week 14, 4 (20%) pts developed csCMVi at median 48 days (range 40-66). Resistance testing performed in 3 of the 4 pts, identified LTV resistance mutations in 2 pts. There were no AEs related to LTV, and none developed EOD. Two pts developed csCMVi in the follow up phase. Three pts died during follow up (due to relapse, treatment related toxicity and GVHD), and four pts are in follow up. Conclusion LTV secondary prophylaxis was safe and prevented recurrent csCMVi in 80% of high risk patients, including patients with prior LTV exposure. Our data supports the utility of LTV for secondary CMV prevention following HCT. Disclosures Boglarka Gyurkocza, MD, Actinium Pharma, Inc. (Grant/Research Support, Research Grant or Support) Genovefa Papanicolaou, MD, ADMA biologics and Siemens Healthineers (Consultant, Other Financial or Material Support)AlloVir (Consultant, Other Financial or Material Support)Amplyx (Scientific Research Study Investigator, Other Financial or Material Support)Astellas (Consultant, Grant/Research Support, Scientific Research Study Investigator, Research Grant or Support, Other Financial or Material Support)Behring (Consultant, Other Financial or Material Support)Chimerix (Consultant, Grant/Research Support, Scientific Research Study Investigator, Research Grant or Support, Other Financial or Material Support)Cidara (Consultant, Other Financial or Material Support)Merck & Co (Consultant, Grant/Research Support, Scientific Research Study Investigator, Research Grant or Support, Other Financial or Material Support)Partners Therapeutics (Consultant, Other Financial or Material Support)Shionogi (Consultant, Other Financial or Material Support)Shire/Takeda (Consultant, Grant/Research Support, Scientific Research Study Investigator, Research Grant or Support, Other Financial or Material Support) Genovefa Papanicolaou, MD, allovir (Individual(s) Involved: Self): Consultant; amplyx (Individual(s) Involved: Self): Consultant; behring (Individual(s) Involved: Self): Consultant; Merck&Co (Individual(s) Involved: Self): Consultant, Investigator and received funding and consulting fees from Merck, Chimerix, Shire and Astellas, Research Grant or Support; octapharma (Individual(s) Involved: Self): Consultant; Partners Therapeutics (Individual(s) Involved: Self): Consultant; takeda (Individual(s) Involved: Self): Consultant, Scientific Research Study Investigator

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