Long-Term Therapy and On-Demand Treatment of Children and Adolescents with Severe Haemophilia A: 12 Years of Experience

1992 ◽  
Vol 22 (5) ◽  
pp. 251-258 ◽  
Author(s):  
H.-H. Brackmann ◽  
H.J. Eickhoff ◽  
J. Oldenburg ◽  
U. Hammersterin
Keyword(s):  
Children And Adolescents ◽  
Term Therapy ◽  
Haemophilia A ◽  
Severe Haemophilia ◽  
Years Of Experience ◽  
On Demand ◽  
Long Term Therapy

Discontinuing early prophylaxis in severe haemophilia leads to deterioration of joint status despite low bleeding rates

2016 ◽  
Vol 115 (05) ◽  
pp. 931-938 ◽  
Author(s):  
Annelies Nijdam ◽  
Wouter Foppen ◽  
Piet de Kleijn ◽  
Evelien P. Mauser-Bunschoten ◽  
Goris Roosendaal ◽  
...  
Keyword(s):  
Functional Limitations ◽  
Objective Assessment ◽  
Haemophilia A ◽  
Severe Haemophilia ◽  
On Demand ◽  
Recommended Treatment ◽  
Objective Monitoring ◽  
Joint Health ◽  
Observational Cohort

SummaryProphylaxis is the recommended treatment for children with severe haemophilia A, but whether prophylaxis should be continued in adulthood is still under debate. Previous studies with limited follow-up have suggested that some patients may be able to stop prophylaxis in adulthood, while maintaining good joint health. This single-centre observational cohort study examined patients with severe haemophilia A born 1970–1988 without inhibitor development, and assessed the long-term consequences of discontinuing prophylaxis. Patient-initiated changes in prophylaxis, including all switches to on-demand treatment lasting a minimum of two consecutive weeks, were recorded from the time self-infusion began until the last evaluation. Sixty-six patients were evaluated at a median age of 32.4 years: 26 of patients had stopped prophylaxis for a median of 10 years, 15 had interrupted prophylaxis and 59 had continued prophylaxis. Annual joint bleeding rate (AJBR), Haemophilia Joint Health Score (HJHS-2.1; 0–124 points), radiological Pettersson score (0–78 points) and Haemophilia Activities List score (HAL; 100–0 points) were compared between patients who stopped and patients who continued prophylaxis. Although self-reported bleeding rates and functional limitations were similar in both groups (AJBR: 1.5 vs 1.2 and HAL: 84 vs 84 for those who stopped and continued prophylaxis, respectively), objective assessment of joint status showed increased arthropathy after 10 years of on-demand treatment in patients who stopped prophylaxis compared with those who continued (HJHS: 23 vs. 14 and Pettersson: 16 vs 5, respectively; P< 0.01). These results support continuation of long-term prophylaxis in adults and demonstrate the need for objective monitoring of joint status.Trial registration: Dutch Trial Registry number 3098; UTN U1111–1121–7069.

Prophylactic versus on-demand treatment strategies for severe haemophilia: a comparison of costs and long-term outcome

Haemophilia ◽  
2002 ◽  
Vol 8 (6) ◽  
pp. 745-752 ◽  
Author(s):  
K. Fischer ◽  
J. G. Van der Bom ◽  
P. Molho ◽  
C. Negrier ◽  
E. P. Mauser-Bunschoten ◽  
...  
Keyword(s):  
Treatment Strategies ◽  
Haemophilia A ◽  
Severe Haemophilia ◽  
Term Outcome ◽  
Long Term Outcome ◽  
On Demand

Benefits of prophylaxis versus on-demand treatment in adolescents and adults with severe haemophilia A: the POTTER study

2015 ◽  
Vol 114 (07) ◽  
pp. 35-45 ◽  
Author(s):  
Giulio Feola ◽  
Angelo Claudio Molinari ◽  
Cristina Santoro ◽  
Gianna Franca Rivolta ◽  
Dorina Bianca Cultrera ◽  
...  
Keyword(s):  
Controlled Trial ◽  
Secondary Prophylaxis ◽  
Annual Number ◽  
Haemophilia A ◽  
Severe Haemophilia ◽  
Open Label ◽  
On Demand ◽  
Adolescents And Adults ◽  
Bleeding Episodes

SummaryRigorous evidence is lacking on long-term outcomes of factor VIII (FVIII) prophylaxis initiated in adolescent or adult patients with severe haemophilia A. The prospective, open-label Prophylaxis versus On-demand Therapy Through Economic Report (POTTER) study (Clinical-Trials.gov NCT01159587) compared long-term late secondary prophylaxis (recombinant FVIII-FS 20–30 IU/kg thrice weekly) with on-demand treatment in patients aged 12 to 55 years with severe haemophilia A. The annual number of joint bleeding episodes (primary endpoint), total bleeding episodes, orthopaedic and radiologic (Pettersson) scores, health-related quality of life (HRQoL), pharmacoeconomic impact, and safety were evaluated over a > 5-year period (2004–2010). Fifty-eight patients were enrolled at 11 centres in Italy; 53 (27 prophylaxis, 26 on demand) were evaluated and stratified into 2 age subgroups (12–25 and 26–55 years). Patients receiving prophylaxis experienced a significantly lower number of joint bleeding episodes vs the on–demand group (annualised bleeding rate, 1.97 vs 16.80 and 2.46 vs 16.71 in younger and older patients, respectively; p=0.0043). Results were similar for total bleeding episodes. Prophylaxis was associated with significantly fewer target joints (p< 0.001), better orthopaedic (p=0.0019) and Pettersson (p=0.0177) scores, better HRQoL, and fewer days of everyday activities lost (p< 0.0001) but required significantly higher FVIII product consumption. The POTTER study is the first prospective, controlled trial documenting long-term benefits of late secondary prophylaxis in adolescents and adults with severe haemophilia A. The benefits of reduced bleeding frequency, improved joint status, and HRQoL may offset the higher FVIII consumption and costs.

Antithrombotic Therapy for DVT/PE

1997 ◽  
Vol 17 (03) ◽  
pp. 161-162
Author(s):  
Thomas Hyers
Keyword(s):  
Molecular Weight ◽  
Low Molecular Weight Heparin ◽  
Oral Anticoagulants ◽  
Cost Effective ◽  
Therapeutic Agents ◽  
Great Promise ◽  
Term Therapy ◽  
Low Molecular Weight ◽  
Long Term Therapy

SummaryProblems with unfractionated heparin as an antithrombotic have led to the development of new therapeutic agents. Of these, low molecular weight heparin shows great promise and has led to out-patient therapy of DVT/PE in selected patients. Oral anticoagulants remain the choice for long-term therapy. More cost-effective ways to give oral anticoagulants are needed.

Office-based post-marketing surveillance study on the influence of long term therapy with aripiprazole in patients with schizophrenia

2007 ◽  
Vol 40 (05) ◽  
Author(s):  
M Kungel ◽  
A Engelhardt ◽  
T Spevakné-Göröcs ◽  
M Ebrecht ◽  
C Werner ◽  
...  
Keyword(s):  
Surveillance Study ◽  
Term Therapy ◽  
Post Marketing Surveillance ◽  
Post Marketing ◽  
Long Term Therapy

scholarly journals Amiodarone-induced thyroid dysfunction in children: insights from the THYRAMIO study

2021 ◽  
Vol 12 ◽  
pp. 204201882110011
Author(s):  
Sarah Montenez ◽  
Stéphane Moniotte ◽  
Annie Robert ◽  
Lieven Desmet ◽  
Philippe A. Lysy
Keyword(s):  
Predictive Value ◽  
Thyroid Dysfunction ◽  
Term Therapy ◽  
Older Children ◽  
Thyroid Status ◽  
Clinical Series ◽  
Amiodarone Treatment ◽  
Long Term Therapy ◽  
Treatment Dosage

Background: Amiodarone treatment is effective against various types of arrhythmias but is associated with adverse effects affecting, among other organs, thyroid function. Amiodarone-induced thyroid dysfunction was not thoroughly evaluated in children as it was in adults, yet this affection may lead to irreversible neurodevelopmental complications. Our study aimed to define the incidence and risk factors of amiodarone-induced thyroid dysfunction in children. Methods: The study was designed as an observational study with a retrospective clinical series of 152 children treated by amiodarone in the Pediatric Cardiology Unit of our center from 1990 to 2019. All patients were divided into three groups according to their thyroid status: euthyroid, AIH (amiodarone-induced hypothyroidism) or AIT (amiodarone-induced thyrotoxicosis). Patients from these three groups were compared in terms of key clinical and therapeutic features. Results: Amiodarone-induced thyroid dysfunction was present in 23% of patients. AIT (5.3%) was three times less common than AIH (17.7%), and its occurrence increased with older age ( p < 0.05), treatment dosage ( p < 0.05), treatment duration ( p < 0.05) and the number of loading doses administered ( p < 0.05). There were no distinctive clinical features between euthyroid and AIH groups. A multivariable prediction model of AIT was built, with a yield of 66.7% as positive predictive value and 96.7% as negative predictive value. Conclusion: We observed that one in five children developed amiodarone-induced thyroid dysfunction. Special attention is required for older children with a high dosage and long-term therapy and who received a large number of loading doses, since these children are at risk to develop AIT, which is more delicate to manage than AIH.

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