scholarly journals Response to: ‘Off-label use of tofacitinib: a potential treatment option for SAPHO syndrome’ by Xie et al

2020 ◽  
pp. annrheumdis-2020-217956
Author(s):  
Yueting Li ◽  
Yihan Cao ◽  
Chen Li ◽  
Wen Zhang
Keyword(s):  
Treatment Option ◽  
Sapho Syndrome ◽  
Potential Treatment ◽  
Off Label Use ◽  
Off Label ◽  
Label Use

A randomized phase II trial of oral vinorelbine as second-line therapy for patients with malignant pleural mesothelioma.

2021 ◽  
Vol 39 (15_suppl) ◽  
pp. 8507-8507
Author(s):  
Dean Anthony Fennell ◽  
Angela Claire Casbard ◽  
Catharine Porter ◽  
Robin Rudd ◽  
Jason Francis Lester ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Treatment Option ◽  
Malignant Pleural Mesothelioma ◽  
Hazard Ratio ◽  
Clinical Activity ◽  
Pleural Mesothelioma ◽  
Off Label Use ◽  
Off Label ◽  
The Impact ◽  
Label Use

8507 Background: All patients with malignant pleural mesothelioma (MPM) eventually relapse following standard chemotherapy. However, there is no standard treatment option in this setting. Vinorelbine, exhibits useful clinical activity but has not been formally evaluated in a randomised clinical trial, despite its widespread off-label use worldwide. BRCA1 regulates spindle assembly checkpoint in MPM and predicts vinorelbine sensitivity in preclinical models [1,2], suggesting that BRCA1 negative patients may be chemoresistant. Methods: VIM, a Cancer Research UK funded, investigator-initiated randomised controlled phase 2 multi-centre UK trial, enrolled patients with MPM who had progressed after first-line chemotherapy. Pts were randomised 2:1 to either vinorelbine (60mg/m2 weekly Q21d escalating to 80mg/m2 from cycle 2) + active supportive care (ASC) versus ASC until disease progression, unacceptable toxicity or withdrawal of consent. The primary outcome was progression free survival (PFS) defined as the time from randomisation to any progression (based on Modified RECIST criteria for assessment of response in malignant pleural mesothelioma) or death. The trial had 90% power to detect a hazard ratio of 0.65 at the one-sided 20% significance level. Secondary endpoints were overall survival (OS), tolerability and safety. Results: Between May 2016 and Oct 2018, 154 patients were recruited from 10 UK sites and randomised to vinorelbine + ASC (n=98) or ASC alone (n=56). In the Intention-to-treat analysis, after 129 events, median PFS was 4.2 months (m) for vinorelbine + ASC compared to 2.8m for ASC alone (Hazard Ratio (HR) 0.59; 95% CI: 0.41 to 0.85; one-sided p = 0.0017). 108 deaths were reported. Median OS was 9.3m for vinorelbine + ASC compared to 9.1m for ASC alone (HR=0.79; 95% CI: 0.53 to 1.17; two-sided p = 0.24). Toxicity data and subgroup analyses including the impact of BRCA1 deficiency will be presented. Conclusion: The trial met its primary endpoint. Vinorelbine demonstrates useful clinical efficacy in relapsed MPM, supporting its off-label use, as a treatment option for patients with relapsed MPM.[1] Busacca et al, J Pathol 2012, 227(2), 200. [2] Busacca et al, Mol Cancer Res, 2021, 20(2) 379. Clinical trial information: NCT02139904.

Peginterferon Alfa 2a: Treatment Option for Coagulopathy Associated with Vascular Malformation?.

Blood ◽  
2005 ◽  
Vol 106 (11) ◽  
pp. 4104-4104
Author(s):  
Charis von Auer ◽  
Manuela Krause ◽  
Wolfgang Miesbach ◽  
Inge Scharrer
Keyword(s):  
Treatment Option ◽  
Clinical Examination ◽  
Interferon Therapy ◽  
Size Reduction ◽  
Bleeding Complications ◽  
Off Label Use ◽  
Laboratory Findings ◽  
Off Label ◽  
Peginterferon Alfa ◽  
Label Use

Abstract We applied peginterferon alfa 2a for off label use therapy in a 44 year old male patient (pat.) with extensive venous malformation (VM) and associated localized intravascular coagulation (LIC). Since birth he had huge VMs in the left leg and left lower abdomen. During surgery and tooth extraction he had severe bleeding complications. In 2003 he reported a sensation of continually growing VMs and requested for therapy. Clinical findings (examination, contrast medium CT) showed no considerable growth of VMs in comparison to former examinations. Laboratory findings were low fibrinogen, low factor VIII activity (FVIII:C) and factor XIII activity (FXIII:C), prolonged aPTT, mild thrombocytopenia and elevated d-dimers. Vascular surgeons disadvised another surgery. LMWH therapy had been carried out before and was ineffective considering coagulopathy and sizereduction of vascular malformations. Considering antiproliferative properties we decided for off label use therapy with peginterferon alfa 2a (Pegasys), 180 μg once a week. Therapy started September 2004 and was applied for 16 weeks. Laboratory tests, clinical examination and photo documentation were done before, during and after therapy. During and after therapy laboratory findings showed normalization of fibrinogen, FVIII:C levels, aPTT and rising FXIII:C levels. We saw no significant change of VMs in clinical examination and photographs. The pat. reported no sense of vascular size reduction but also no sense of growing malformations. Due to interferon side effects such as lowered blood count, therapy was finished after 16 weeks. In conclusion the LIC in the VMs seemed to improve under interferon therapy with normalization of fibrinogen and FVIII:C. Even though there was no significant size reduction of VMs yet, this therapy could be an important treatment option for normalization of coagulation parameters e.g. before surgery. In our patient interferon therapy seemed to be more effective than LMWH therapy. In literature only heparin was reported to be effective in coagulopathies associated with VMs, not interferon. Therefore the positive laboratory findings in our patient are remarkable.

Indikationsfremde Anwendung von Medikamenten und Medizinprodukten in der Notfallmedizin

2019 ◽  
Vol 14 (04) ◽  
pp. 361-371
Author(s):  
Karl Peter Ittner ◽  
Joachim Koppenberg ◽  
Ute Walter
Keyword(s):  
Off Label Use ◽  
Off Label ◽  
Label Use

ZusammenfassungWenn zugelassene Arzneimittel außerhalb der in der entsprechenden Fachinformation dargelegten Beschreibungen angewendet werden, dann spricht man von einer nicht zulassungskonformen Anwendung oder von einem Off-Label-Use. Wie in fast allen medizinischen Fachgebieten gibt es auch im Rettungsdienst sogenannte Off-Label-Use-Pharmakotherapien. Sofern evidenzbasierte Informationen zu einer nicht zulassungskonformen Anwendung vorliegen, und insbesondere im konkreten Notfall keine zulassungskonforme Möglichkeit besteht, dann ist diese gerechtfertigt. Verwendet ein Notarzt aber ein Medizinprodukt außerhalb der Zulassung, dann stellt er ein neues Produkt her und haftet persönlich bei einem Patientenschaden.

Botulinumtoxin in der Urologie

2010 ◽  
Vol 29 (09) ◽  
pp. 551-555
Author(s):  
W. N. Vance ◽  
J. Wissel
Keyword(s):  
Off Label Use ◽  
Botulinumtoxin A ◽  
Off Label ◽  
Label Use

ZusammenfassungAlle Indikationen zur Anwendung von Botulinumtoxin A (BoNT A) in der Urologie befinden sich im Status des sogenannten off label use, entsprechend sind Kernfragen wie z. B. die Kostenübernahme nicht geklärt. Erst 20 Jahre nach der ersten Anwendung in der Urologie werden Zulassungsstudien durchgeführt. Andererseits sind insbesondere die Behandlungsmöglichkeiten im Bereich der neurogenen Harnblase so etabliert, dass sie bereits in die urologischen Leitlinien Einzug gefunden haben. Hinsichtlich der Dosierung von BoNT A und der optimalen Anwendungstechnik bestehen weder allgemein anerkannte Handlungsanweisungen noch offizielle Empfehlungen. Dies gilt auch im Bereich der Anästhesie zur Injektion von BoNT A im urologischen Gebiet. Nicht wenige Patienten schrecken davor zurück, sich regelmäßig, zum Teil jährlich, einer Allgemeinanästhesie zu unterziehen. Ein besonderer Hoffnungsschimmer stellt für multimorbide Patienten mit hohem OP-Risiko die sich entwickelnde Behandlung der Prostatahyperplasie mittels BoNT A dar, insbesondere da in allen Studien die Lokalanästhesie angewandt wurde und nur wenige Nebenwirkungen auftraten.

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