Lack of long-term follow-up after paediatric-adult transition in coeliac disease is not associated with complications, ongoing symptoms or dietary adherence

Background Follow-up of coeliac disease is recommended to prevent complications associated with unsuccessful treatment. Objective The objective of this article is to evaluate the implementation and significance of long-term follow-up. Methods Medical data were collected from 585 and follow-up questionnaires sent to 559 current adult coeliac disease patients diagnosed in childhood. Diagnostic features and adulthood health outcomes were compared between those with and without adulthood follow-up. Results Of paediatric patients, 92% were followed up 6–24 months after diagnosis. A total of 235 adults responded to the questionnaires a median of 18 years after diagnosis, and 25% of them reported regular follow-up. They were diagnosed more recently than those without follow-up (median year 2001 vs 1995, p = 0.001), being otherwise comparable at diagnosis. Those with follow-up were less often smokers (5% vs 16%, p = 0.042) and relatives of coeliac patients (48% vs 66%, p = 0.018), and more often students (48% vs 28%, p = 0.005) and type 1 diabetics (19% vs 4%, p = 0.001). Lack of follow-up was not associated with complications, ongoing symptoms, poorer general health or dietary adherence. All completely non-adherent patients were without follow-up. Conclusions Most coeliac disease patients diagnosed in childhood were not followed up according to recommendations in adulthood. The individual effect of this on long-term treatment outcomes varied markedly.

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Child–Adult Transition in Sarcoidosis: A Series of 52 Patients

Journal of Clinical Medicine ◽

10.3390/jcm9072097 ◽

2020 ◽

Vol 9 (7) ◽

pp. 2097 ◽

Cited By ~ 1

Author(s):

Simon Chauveau ◽

Florence Jeny ◽

Marie-Emeline Montagne ◽

Rola Abou Taam ◽

Véronique Houdouin ◽

...

Keyword(s):

Median Duration ◽

Severe Disease ◽

Clinical Information ◽

Treatment Interruption ◽

Term Treatment ◽

Adult Transition ◽

Long Term Treatment ◽

Pediatric Onset

(1) Background: Pediatric sarcoidosis is a rare and mostly severe disease. Very few pediatric series with a prolonged follow-up are reported. We aimed to evaluate the evolution of pediatric sarcoidosis in adulthood. (2) Material and methods: Patients over 18-years-old with a pediatric-onset sarcoidosis (≤15-year-old) who completed at least a three-year follow-up in French expert centers were included. Clinical information at presentation and outcome in adulthood were studied. (3) Results: A total of 52 patients were included (34 prospectively in childhood and 18 retrospectively in adulthood), with a mean age of 12 (±2.7) at diagnosis. The median duration time of follow-up was 11.5 years (range 3–44.5). Relapses mostly occurred during treatment decrease (84.5%), others within the three years after treatment interruption (9.1%), and rarely when the disease was stable for more than three years (6.4%). Sarcoidosis was severe in 11 (21.2%) in adulthood. Patients received a high corticosteroid cumulative dose (median 17,900 mg) for a median duration of five years (range 0–32), resulting in mostly mild (18; 35.3%) and rarely severe (2; 3.8%) adverse events. (4) Conclusions: Pediatric-onset sarcoidosis needed a long-term treatment in almost half of the patients. Around one fifth of pediatric-onset sarcoidosis patients had severe sarcoidosis consequences in adulthood.

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United States open-label glatiramer acetate extension trial for relapsing multiple sclerosis: MRI and clinical correlates

Multiple Sclerosis Journal ◽

10.1177/135245850100700107 ◽

2001 ◽

Vol 7 (1) ◽

pp. 33-41 ◽

Cited By ~ 66

Author(s):

Jerry S Wolinsky ◽

Ponnada A Narayana ◽

Kenneth P Johnson ◽

Keyword(s):

Glatiramer Acetate ◽

Term Treatment ◽

Open Label ◽

Clinical Correlates ◽

Long Term Treatment ◽

Open Label Extension ◽

Long Term Follow Up ◽

Relapsing Multiple Sclerosis

After the placebo-controlled extension of the pivotal US trial of glatiramer acetate for the treatment of relapsing multiple sclerosis ended, 208 participants entered an open-label, long-term treatment protocol. Magnetic resonance imaging (MRI) was added to the planned evaluations of these subjects to determine the consequences of long-term treatment on MRI-defined pathology and evaluate its clinical correlates. Of the 147 subjects that remained on long-term follow-up, adequate images were obtained on 135 for quantitative MRI analysis. The initial imaging sessions were performed between June 1998 and January 1999 at 2447+61 days (mean+standard deviation) after the subject's original randomization. Clinical data from a preplanned clinical visit were matched to MRI within 3+51 days. At imaging, 66 patients originally randomized to placebo (oPBO) in the pivotal trial had received glatiramer acetate for 1476+63 days, and 69 randomized to active treatment with glatiramer acetate (oGA) were on drug for 2433+59 days. The number of documented relapses in the 2 years prior to entering the open-label extension was higher in the group originally randomized to placebo (oPBO=1.86+1.78, oGA=1.03+1.28; P=0.002). The annualized relapse rate observed during the open-label study was similar for both groups (oPBO=0.27, +0.45 oGA=0.28+0.40), but the reduction in rate from the placebo-controlled phase was greater for those beginning therapy with GA (oPBO reduced by 0.66+0.71, oGA reduced by 0.23+0.58; P=0.0002). One or more gadolinium enhancing lesions were found in 27.4% of all patients (number of distinct enhancements=1.16+2.52, total enhanced tissue volume=97+26 ml). The risk of having an enhancement was higher in those with relapses during the open-label extension (odds ratio 4.65, 95% confidence interval (CI) 2.0 to 10.7; P=0.001). The odds for finding an enhancement was 2.5 times higher for those patients originally randomized to placebo (CI 1.1 to 5.4; P=0.02) compared to those always on glatiramer acetate. MRI-metrics indicative of chronic pathology, particularly measures of global cerebral tissue loss (atrophy), were uniformly worse for those originally on placebo. These observations enrich our long-term follow up of the clinical consequences of treatment with glatiramer acetate to include its apparent effects on MRI-defined pathology. They show that the effect of glatiramer acetate on enhancements is definite, but modest, consistent with the drug's described mechanisms of action, and that a delay in initiating treatment results in progression of MRI-measured pathology that can be prevented.

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Joint-preserving surgical treatment options for irreparable posterosuperior rotator cuff tear

Obere Extremität ◽

10.1007/s11678-021-00657-6 ◽

2021 ◽

Author(s):

Florian Grubhofer ◽

Jon JP Warner

Keyword(s):

Rotator Cuff ◽

Treatment Outcomes ◽

Treatment Options ◽

Term Treatment ◽

Therapeutic Options ◽

Rotator Cuff Tears ◽

Long Term Treatment ◽

Long Term Follow Up

AbstractTreatment of irreparable rotator cuff tears in young active patients is challenging. A variety of therapeutic options are available. Only a few joint-preserving treatment options show reliable improvements over a long-term follow-up period. However, the treatment outcomes of joint preservation procedures are not comparable to those of RTSA, as patients are typically younger and have higher expectations. It is remarkable that most of the joint-preserving therapeutic options for irreparable rotator cuff ruptures lack long-term treatment results. This article highlights the indications, technical aspects, and treatment outcomes of the most commonly performed joint-preserving surgeries for irreparable rotator cuff rupture.

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Advances in the Treatment of Anal Fistula: A Mini-Review of Recent Five-Year Clinical Studies

Frontiers in Surgery ◽

10.3389/fsurg.2020.586891 ◽

2021 ◽

Vol 7 ◽

Author(s):

Lijiang Ji ◽

Yang Zhang ◽

Liang Xu ◽

Jun Wei ◽

Liping Weng ◽

...

Keyword(s):

Anal Fistula ◽

Term Treatment ◽

Large Sample Size ◽

Effective Strategies ◽

Long Term Treatment ◽

Long Term Follow Up ◽

Pros And Cons ◽

Fistula Treatment

Anal fistula, with its complicated pathogenesis, has been considered as a clinical challenge for centuries. The risk of frequent recurrence and incontinence constitutes a considerable threat in the long-term treatment of anal fistula. In this work, we narratively reviewed the scientific literature of new techniques that have been used for anal fistula treatment over the recent 5 years, objectively evaluated the pros and cons of each technique on the basis of clinical outcomes, and tried to disclose the effective strategies for anal fistula treatment. Up to date, surgery is the main method used for treating anal fistula, but there is no simple technique that can completely heal complex anal fistula. In the course of surgery treatment, the healing outcome, and the protection of anal function should be weighed comprehensively. Among the innovative techniques that have emerged in recent years, combined techniques based on drainage Seton and LIFT-plug seem to be the relatively effective therapies, but their effectiveness requires more multi-center prospective randomized controlled trials with large sample size and long-term follow-up to be validated.

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Long-term efficacy and safety with ibrutinib (ibr) in previously treated chronic lymphocytic leukemia (CLL): Up to four years follow-up of the RESONATE study.

Journal of Clinical Oncology ◽

10.1200/jco.2017.35.15_suppl.7510 ◽

2017 ◽

Vol 35 (15_suppl) ◽

pp. 7510-7510 ◽

Cited By ~ 9

Author(s):

John C. Byrd ◽

Peter Hillmen ◽

Susan Mary O'Brien ◽

Jacqueline Claudia Barrientos ◽

Nishitha M. Reddy ◽

...

Keyword(s):

Atrial Fibrillation ◽

Term Treatment ◽

Lymphocytic Leukemia ◽

Major Hemorrhage ◽

Prior Therapy ◽

Long Term Treatment ◽

Long Term Follow Up ◽

Previously Treated

7510 Background: Ibr, a first-in-class, once-daily inhibitor of Bruton’s tyrosine kinase, is FDA-approved for all pts with CLL/SLL. We report updated safety and efficacy results with up to 4 y follow-up from the ph III RESONATE trial of ibr vs ofatumumab (ofa). Methods: Pts had ≥1 prior therapy. Pts received 420 mg ibr PO until PD or ofa up to 24 wks. At interim analysis (median 9 mo follow-up), the DMC declared superiority of ibr vs ofa for PFS and OS, and ibr access was recommended for all ofa pts. Long-term follow-up efficacy endpoints are per investigator assessment. Ofa pts were censored at crossover for OS. Results: 391 pts were randomized to receive ibr (n = 195) or ofa (n = 196). Median age was 67 y (40% ≥70 y); 57% had Rai stage III/IV. With median follow-up of 44 mo (53 mo max) for ibr arm, PFS was significantly longer for ibr vs ofa (median NR vs 8 mo, [HR 0.133; P< 0.0001]; 3-y PFS 59% vs 3%) with significant benefit across subgroups. PFS with ibr for del11q subgroup trended to have the most favorable outcome; however, PFS was not statistically different for pts with del17p or del11q or without these FISH abnormalities. At analysis, with the majority of pts (68%) randomized to ofa crossing over to ibr, OS was longer for ibr vs ofa (median OS NR for either arm). The OS rate for ibr at 3 y was 74%. ORR for ibr was 91% with CR/CRi rates (now 9%) increasing over time. Baseline cytopenias improved with extended ibr therapy for hemoglobin (85%), platelet (95%), and absolute neutrophil counts (95%). AE profile of ibr was consistent with previous reports. Major hemorrhage, Gr ≥3 atrial fibrillation, and Gr ≥3 hypertension occurred in 6%, 6%, and 8% of pts, respectively, over a follow-up of up to 4 y. Incidence of most Gr ≥3 AEs decreased from y 1 vs y 2-3: neutropenia- 18% vs 8%; pneumonia- 11% vs 4%; atrial fibrillation- 4% vs 2%, respectively. Discontinuations were most frequently PD (27%) and AE (12%). At analysis, 90 ibr pts (46%) continue ibr on study. Conclusions: Long-term treatment with ibr in this international ph III RESONATE study is tolerable and continues to show sustained PFS and OS regardless of high-risk cytogenetics. Ph III results in relapsed del17p and del11q pts compare favorably to prior ph II reports. Clinical trial information: NCT01578707.

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Long-term treatment effect in cerebrotendinous xanthomatosis depends on age at treatment start

Neurology ◽

10.1212/wnl.0000000000006731 ◽

2018 ◽

Vol 92 (2) ◽

pp. e83-e95 ◽

Cited By ~ 14

Author(s):

Bianca M.L. Stelten ◽

Hidde H. Huidekoper ◽

Bart P.C. van de Warrenburg ◽

Eva H. Brilstra ◽

Carla E.M. Hollak ◽

...

Keyword(s):

Term Treatment ◽

Cerebrotendinous Xanthomatosis ◽

Neurologic Symptoms ◽

Screening Programs ◽

Treatment Start ◽

Long Term Treatment ◽

Disability Status ◽

Long Term Follow Up

ObjectiveTo evaluate the effect of chenodeoxycholic acid treatment on disease progression in cerebrotendinous xanthomatosis (CTX).MethodsIn this retrospective cohort study, we report the clinical long-term follow-up characteristics of 56 Dutch patients with CTX. Age at diagnosis was correlated with clinical characteristics and with the course of modified Rankin Scale (mRS) and Expanded Disability Status Scale (EDSS) scores at follow-up.ResultsMedian follow-up time was 8 years (6 months–31.5 years). Patients diagnosed and treated before the age of 24 years had a significantly better outcome at follow-up. When considering only patients with a good treatment adherence (n = 43), neurologic symptoms, if present, disappeared in all patients who were diagnosed before the age of 24 and treated since. Furthermore, treatment prevented the development of new neurologic symptoms during follow-up. In contrast, 61% of the patients diagnosed and treated after the age of 24 showed deterioration of the neurologic symptoms, with parkinsonism as a treatment-resistant feature. There was an improvement or stabilization in favor of patients diagnosed and treated before the age of 24 compared to those treated after the age of 24: 100% vs 58% for mRS scores and 100% vs 50% for EDSS scores, respectively.ConclusionsTreatment start at an early age can reverse and even prevent the development of neurologic symptoms in CTX. This study emphasizes the importance of early diagnosis in CTX and provides a rationale to include CTX in newborn screening programs.

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