Advertisement

Pharmacovigilance in a rare disease: example of the VIGIAPATH program in pulmonary arterial hypertension

  • Marie-Camille Chaumais
  • Caroline O’Connell
  • Laurent Savale
  • Christophe Guignabert
  • Frédéric Perros
  • Xavier Jaïs
  • Olivier Sitbon
  • Marc Humbert
  • David Montani
Commentary
  • 20 Downloads

Abstract

Spontaneous reporting is the primary method used in pharmacovigilance (PV) to detect drug safety signal. Specific criteria used in pharmacovigilance to prove accountability of a drug are rarely present in rare disease. The low number of alerts also makes it challenging. The aim of this commentary is to raise awareness among pharmacists on issues and opportunities for pharmacovigilance in rare diseases, taking pulmonary arterial hypertension (PAH) as example, from which a subset of cases are drug-induced. It is demonstrated how a dedicated program named VIGIAPATH created to reinforce pharmacovigilance of drug-induced pulmonary arterial hypertension at a national level, led to increase self-reporting and confirm safety signals. Thanks to a specific program such as VIGIAPATH, pharmacists can play an important role in communication with clinicians, patients and regulatory agencies, facilitating the detection of potential safety signals at an early stage in rare disease.

Keywords

Rare diseases Pulmonary arterial hypertension Adverse drug reaction Pharmacovigilance 

Notes

Acknowledgements

The authors thank the French pulmonary hypertension pharmacovigilance network, VIGIAPATH, supported by the Agence Nationale de Sécurité du Médicament et des Produits de Santé (ANSM), the patient association HTaPFrance and acknowledge the contribution of all healthcare providers from the French PAH Network with special thanks to Florence Parent, Gérald Simonneau and all members of the French Reference Center for Pulmonary Hypertension, Hôpital Bicêtre, Assistance Publique Hôpitaux de Paris, Le Kremlin Bicêtre, France.

Funding

Development of the VIGIAPATH program was funded by the Agence Nationale de Sécurité du Médicament et des Produits de Santé (ANSM). This commentary is not funded.

Conflicts of interest

None.

References

  1. 1.
    European Medicines Agency. Guideline on good pharmacovigilance practices (GVP) Module IX—Signal management [Internet]. EMA; 2012 Jun. http://www.ema.europa.eu/ema/index.jsp?curl=pages/regulation/document_listing/document_listing_000345.jsp#section2.
  2. 2.
    D’Alonzo GE, Barst RJ, Ayres SM, Bergofsky EH, Brundage BH, Detre KM, et al. Survival in patients with primary pulmonary hypertension. Results from a national prospective registry. Ann Intern Med. 1991;115:343–9.CrossRefPubMedGoogle Scholar
  3. 3.
    Peacock AJ, Murphy NF, McMurray JJV, Caballero L, Stewart S. An epidemiological study of pulmonary arterial hypertension. Eur Respir J. 2007;30:104–9.CrossRefPubMedGoogle Scholar
  4. 4.
    Galiè N, Humbert M, Vachiery J-L, Gibbs S, Lang I, Torbicki A, et al. 2015 ESC/ERS Guidelines for the diagnosis and treatment of pulmonary hypertension: the Joint Task Force for the Diagnosis and Treatment of Pulmonary Hypertension of the European Society of Cardiology (ESC) and the European Respiratory Society (ERS): Endorsed by: Association for European Paediatric and Congenital Cardiology (AEPC), International Society for Heart and Lung Transplantation (ISHLT). Eur Respir J. 2015;46:903–75.CrossRefPubMedGoogle Scholar
  5. 5.
    Gurtner HP. Aminorex and pulmonary hypertension. A review. Cor Vasa. 1985;27:160–71.PubMedGoogle Scholar
  6. 6.
    Brenot F, Herve P, Petitpretz P, Parent F, Duroux P, Simonneau G. Primary pulmonary hypertension and fenfluramine use. Br Heart J. 1993;70:537–41.CrossRefPubMedPubMedCentralGoogle Scholar
  7. 7.
    Abenhaim L, Moride Y, Brenot F, Rich S, Benichou J, Kurz X, et al. Appetite-suppressant drugs and the risk of primary pulmonary hypertension. International primary pulmonary hypertension study group. N Engl J Med. 1996;335:609–16.CrossRefPubMedGoogle Scholar
  8. 8.
    Savale L, Chaumais M-C, Cottin V, Bergot E, Frachon I, Prevot G, et al. Pulmonary hypertension associated with benfluorex exposure. Eur Respir J. 2012;40:1164–72.CrossRefPubMedGoogle Scholar
  9. 9.
    Montani D, Bergot E, Günther S, Savale L, Bergeron A, Bourdin A, et al. Pulmonary arterial hypertension in patients treated by dasatinib. Circulation. 2012;125:2128–37.CrossRefPubMedGoogle Scholar
  10. 10.
    Savale L, Chaumais M-C, Sitbon O, Humbert M. Pulmonary arterial hypertension in patients treated with interferon. Eur Respir J. 2015;46:1851–3.CrossRefPubMedGoogle Scholar
  11. 11.
    Weatherald J, Chaumais M-C, Savale L, Jaïs X, Seferian A, Canuet M, et al. Long-term outcomes of dasatinib-induced pulmonary arterial hypertension: a population-based study. Eur Respir J. 2017;50:1700217.CrossRefPubMedGoogle Scholar
  12. 12.
    Daccord C, Letovanec I, Yerly P, Bloch J, Ogna A, Nicod LP, et al. First histopathological evidence of irreversible pulmonary vascular disease in dasatinib-induced pulmonary arterial hypertension. Eur Respir J. 2018;51:1701694.CrossRefPubMedGoogle Scholar
  13. 13.
    Riou M, Seferian A, Savale L, Chaumais M-C, Guignabert C, Canuet M, et al. Deterioration of pulmonary hypertension and pleural effusion with bosutinib following dasatinib lung toxicity. Eur Respir J. 2016;48:1517–9.CrossRefPubMedGoogle Scholar
  14. 14.
    Hickey PM, Thompson AAR, Charalampopoulos A, Elliot CA, Hamilton N, Kiely DG, et al. Bosutinib therapy resulting in severe deterioration of pre-existing pulmonary arterial hypertension. Eur Respir J. 2016;48:1514–6.CrossRefPubMedGoogle Scholar
  15. 15.
    Quilot F-M, Georges M, Favrolt N, Beltramo G, Foignot C, Grandvuillemin A, et al. Pulmonary hypertension associated with ponatinib therapy. Eur Respir J. 2016;47:676–9.CrossRefPubMedGoogle Scholar
  16. 16.
    Chabrol A, Mayenga M, Hamid AM, Friard S, Salvator H, Doubre H, et al. Lorlatinib - Induced pulmonary arterial hypertension. Lung Cancer. 2018;120:60–1.CrossRefPubMedGoogle Scholar
  17. 17.
    Guignabert C, Phan C, Seferian A, Huertas A, Tu L, Thuillet R, et al. Dasatinib induces lung vascular toxicity and predisposes to pulmonary hypertension. J Clin Invest. 2016;126:3207–18.CrossRefPubMedPubMedCentralGoogle Scholar
  18. 18.
    Ranchoux B, Günther S, Quarck R, Chaumais M-C, Dorfmüller P, Antigny F, et al. Chemotherapy-induced pulmonary hypertension: role of alkylating agents. Am J Pathol. 2015;185:356–71.CrossRefPubMedGoogle Scholar
  19. 19.
    Perros F, Günther S, Ranchoux B, Godinas L, Antigny F, Chaumais M-C, et al. Mitomycin-Induced pulmonary veno-occlusive disease: evidence from human disease and animal Models. Circulation. 2015;132:834–47.CrossRefPubMedGoogle Scholar
  20. 20.
    Belge C, Quarck R, Delcroix M. Letter by Belge et al Regarding Article, “Mitomycin-induced pulmonary veno-occlusive disease: evidence from human disease and animal models.” Circulation. 2016;133:e591.Google Scholar
  21. 21.
    Savale L, Chaumais M-C, Dorfmuller P, Humbert M, Montani D. Lung transplantation for mitomycin-induced pulmonary veno-occlusive disease. Presse Med Paris Fr. 1983;2017(46):1223–5.Google Scholar
  22. 22.
    Orcholski ME, Yuan K, Rajasingh C, Tsai H, Shamskhou EA, Dhillon NK, et al. Drug-induced pulmonary arterial hypertension: a primer for clinicians and scientists. Am J Physiol Lung Cell Mol Physiol. 2018;314:L967–83.CrossRefPubMedGoogle Scholar
  23. 23.
    Toklu HZ, Mensah E. Why do we need pharmacists in pharmacovigilance systems? Online J Public Health Inform. 2016;8:e193.CrossRefPubMedPubMedCentralGoogle Scholar

Copyright information

© Springer Nature Switzerland AG 2018

Authors and Affiliations

  1. 1.Faculté de PharmacieUniv. Paris-Sud, Université Paris-SaclayChatenay MalabryFrance
  2. 2.AP-HP, Service de pharmacie, DHU Thorax InnovationHôpital Antoine BéclèreClamartFrance
  3. 3.INSERM UMR_S 999Hôpital Marie LannelongueLe Plessis RobinsonFrance
  4. 4.Centre de Recherche de l’Institut Universitaire de Cardiologie et de Pneumologie de QuébecLaval UniversityQuebec CityCanada
  5. 5.Faculté de MédecineUniversité Paris-Saclay, Univ. Paris–SudLe Kremlin BicêtreFrance
  6. 6.AP-HP, Service de Pneumologie, centre de référence de l’hypertension pulmonaire, DHU Thorax InnovationHôpital BicêtreLe Kremlin BicêtreFrance

Personalised recommendations