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Treatment Patterns And Outcomes In Patients Treated With Benefix Or Refacto/Refacto AF - A Swedish Cohort Study

30 stycznia 2019 zaktualizowane przez: Pfizer

TREATMENT PATTERNS AND OUTCOMES IN PATIENTS TREATED WITH BENEFIX OR REFACTO/REFACTO AF - A SWEDISH COHORT STUDY

The overall aim of the study is to describe demographic and clinical characteristics, treatment patterns and outcomes, in the populations of hemophilia patients treated with BeneFIX and ReFacto/ReFacto AF in Sweden

Przegląd badań

Status

Zakończony

Warunki

Szczegółowy opis

The overall aim of the study is to describe demographic and clinical characteristics, treatment patterns and outcomes, as well as the related direct treatment costs in the populations of hemophilia patients treated with BeneFIX and ReFacto/ReFacto AF, and in subgroups (e.g. level of severity) at the MHC in Sweden.

The study population will consist of all patients diagnosed with haemophilia (D66.9 (haemophilia A) D67.9 (haemophilia B) in International Statistical Classification of Diseases and Related Health Problems (ICD-10) that have been registered in the MHR since 1977 and that have had at least one registered prescription of BeneFIX or ReFacto/ReFacto AF in the MHR since market authorization of the respective product (Benefix August 27 1997, ReFacto April 13 1999, ReFacto AF July 1 2009). Diseased individuals are included. Information on drugs picked up at the pharmacy is available in the Prescribed Drug Register from 2005.

Typ studiów

Obserwacyjny

Zapisy (Rzeczywisty)

86

Kryteria uczestnictwa

Badacze szukają osób, które pasują do określonego opisu, zwanego kryteriami kwalifikacyjnymi. Niektóre przykłady tych kryteriów to ogólny stan zdrowia danej osoby lub wcześniejsze leczenie.

Kryteria kwalifikacji

Wiek uprawniający do nauki

  • Dziecko
  • Dorosły
  • Starszy dorosły

Akceptuje zdrowych ochotników

Nie

Metoda próbkowania

Próbka prawdopodobieństwa

Badana populacja

The study population will consist of all patients diagnosed with hemophilia (D66.9 (hemophilia A) D67.9 (hemophilia B) in International Statistical Classification of Diseases and Related Health Problems (ICD-10) that have been registered in the Malmö Hemophilia Register since 1977 and that have had at least one registered prescription of BeneFIX or ReFacto/ReFacto AF in the MHR since market authorization of the respective product (Benefix August 27 1997, ReFacto April 13 1999, ReFacto AF July 1 2009). Diseased individuals are included.

Opis

Inclusion Criteria:

  • The study population will consist of all patients diagnosed with hemophilia (D66.9 (hemophilia A) D67.9 (hemophilia B) in International Statistical Classification of Diseases and Related Health Problems (ICD-10) that have been registered in the Malmö Hemophilia Register since 1977 and that have had at least one registered prescription of BeneFIX or ReFacto/ReFacto AF in the MHR since market authorization of the respective product

Exclusion Criteria:

  • No exclusion criteria in this study

Plan studiów

Ta sekcja zawiera szczegółowe informacje na temat planu badania, w tym sposób zaprojektowania badania i jego pomiary.

Jak projektuje się badanie?

Szczegóły projektu

Kohorty i interwencje

Grupa / Kohorta
Patients with Haemophilia A
Patients in The MHR diagnosed with Haemophilia A
Patients with Haemophilia B
Patients in The MHR diagnosed with Haemophilia B

Co mierzy badanie?

Podstawowe miary wyniku

Miara wyniku
Opis środka
Ramy czasowe
Age of Participants at Disease Diagnosis
Ramy czasowe: At disease diagnosis within 11 years before the study start date (Day 1)
At disease diagnosis within 11 years before the study start date (Day 1)
Age of Participants at Start of Treatment With Benefix or Refacto
Ramy czasowe: At start of treatment with Benefix/Refacto within 11 years before the study start date (Day 1)
At start of treatment with Benefix/Refacto within 11 years before the study start date (Day 1)
Age of Participants at Start of Replacement Treatment
Ramy czasowe: At start of replacement treatment within 11 years before the study start date (Day 1)
Participants diagnosed with coagulation disorders received intravenous replacement treatment of the missing coagulation factor. Replacement treatment could be given prophylactically or to stop the bleed and/or to stop it from becoming more severe.
At start of replacement treatment within 11 years before the study start date (Day 1)
Average Prescribed Dose Per Infusion for Factor VIII and Factor IX Concentrates
Ramy czasowe: For the duration of 11 years before the study start date (Day 1)
Participants diagnosed with coagulation disorders received intravenous replacement treatment of the missing coagulation factor (Factor VIII or IX). Data was measured in international units (IU) for factor VIII and factor IX concentrates.
For the duration of 11 years before the study start date (Day 1)
Average Prescribed Dose Per Kilogram Bodyweight for Factor VIII and Factor IX Concentrates
Ramy czasowe: For the duration of 11 years before the study start date (Day 1)
The average dose per kilogram body weight of factor VIII and IX was defined as a set of two derived variables from the MHR based on information on prescribed replacement treatment (Benefix or Refacto/Refacto AF): prescribed dose per infusion divided by registered body weight (all participants) and prescribed dose per week (prescribed dose per infusion multiplied by registered number of infusions per week) divided by registered body weight (only participants on prophylaxis).
For the duration of 11 years before the study start date (Day 1)
Percentage of Participants on Prophylactic Treatment
Ramy czasowe: For the duration of 11 years before the study start date (Day 1)
Prophylactic treatment was defined as administration of drug regularly to reduce the insufficiency of coagulation factor to prevent bleeding to occur.
For the duration of 11 years before the study start date (Day 1)
Average Prescribed Frequency of Infusions Per Week Dispensed for Haemophilia A or B Participants With Prophylaxis
Ramy czasowe: For the duration of 11 years before the study start date (Day 1)
For the duration of 11 years before the study start date (Day 1)
Average Prescribed Annual Dose of Factor VIII and IX Concentrates for Participants on Prophylaxis
Ramy czasowe: For the duration of 11 years before the study start date (Day 1)
Participants diagnosed with coagulation disorders received intravenous replacement treatment of the missing coagulation factor (Factor VIII or IX). Prescribed annual dose of factor concentrate was derived from the MHR registration of prescribed annual dose of factor concentrate.
For the duration of 11 years before the study start date (Day 1)
Average Prescribed Annual Dose of Factor VIII and IX Concentrates Per Kilogram Bodyweight for Participants on Prophylaxis
Ramy czasowe: For the duration of 11 years before the study start date (Day 1)
Participants diagnosed with coagulation disorders received intravenous replacement treatment of the missing coagulation factor (Factor VIII or IX). Prescribed annual dose of factor concentrate per kilogram body weight was derived from the MHR registration of prescribed annual dose of factor concentrate and the registered body weight.
For the duration of 11 years before the study start date (Day 1)
Average Annual Registered Consumption of Factor VIII and IX Concentrates
Ramy czasowe: For the duration of 11 years before the study start date (Day 1)
Participants diagnosed with coagulation disorders received intravenous replacement treatment of the missing coagulation factor (Factor VIII or IX). Average annual registered consumption of factor VIII and IX concentrates was defined as MHR registered participant's reports on factor concentrate consumption during calendar year.
For the duration of 11 years before the study start date (Day 1)
Number of Participants With Consumption of Factor VIII, Factor IX, Factor rVIIa and/or aPCC Concentrate
Ramy czasowe: For the duration of 11 years before the study start date (Day 1)
Participants who develop inhibitors to factor VIII or IX concentrates were treated with bypassing agents in the form of activated prothrombin complex concentrate (aPCC) which was measured in units (U) and/or recombinant factor VIIa (rFVIIa) measured in micrograms (mcg).
For the duration of 11 years before the study start date (Day 1)
Percentage of Time on Refacto or Benefix
Ramy czasowe: For the duration of 11 years before the study start date (Day 1)
Percentage of time (in days) on Refacto/Refacto AF or Benefix was calculated as total number of days when participant was prescribed Refacto/Refacto AF or Benefix over the total number of days on any replacement treatment. Total number of days on any replacement treatment was derived from date of start of replacement treatment according to MHR and August 31, 2015 or date of death, whichever was earliest.
For the duration of 11 years before the study start date (Day 1)
Average Use of Factor Concentrate Per Surgery Event at Hospital for Invasive Procedures
Ramy czasowe: For the duration of 11 years before the study start date (Day 1)
For the duration of 11 years before the study start date (Day 1)
Average Factor Concentrate Use at Hospital for Invasive Procedures
Ramy czasowe: For the duration of 11 years before the study start date (Day 1)
Average factor concentrate use was calculated as percentage of total annual use of factor concentrates (in IU) at hospital for invasive procedures.
For the duration of 11 years before the study start date (Day 1)
Average Annual Number of Filled Prescriptions of Factor Concentrate
Ramy czasowe: For a duration of 3 years (for up to 11 years before the study start Day 1)
Data at end of every year was based on the number of filled prescriptions during the last 3 years.
For a duration of 3 years (for up to 11 years before the study start Day 1)
Average Annual Number of Dispensed Units of Factor Concentrate
Ramy czasowe: For a duration of 3 years (for up to 11 years before the study start Day 1)
To assess annual consumption of factor concentrates, calculations were based on prescription date as start of use and the day before the next prescription as the last date of use of the factor concentrates retrieved. The annual number of dispensed units of factor concentrate were then summed up of all dispensed units of factor concentrate with periods within the calendar year plus estimates of average daily use periods extending over two years. Data at end of every year was based on the number of dispensed units of factor concentrate during the last 3 years.
For a duration of 3 years (for up to 11 years before the study start Day 1)
Average Number of Units of Benefix or Refacto
Ramy czasowe: For the duration of 11 years before the study start date (Day 1)
Data from the National Board of Health and Welfare (NBHW) had information on all filled prescriptions from July 1, 2005-last date of observation (August 31, 2015). Derived variable calculated as total number of units of filled prescriptions of Refacto/Refacto AF and Benefix over the total units of all factor VIII concentrates and factor IX concentrates, respectively. Total number of units were derived from the date of start of NBHW to last available observation or date of death, whichever was earliest.
For the duration of 11 years before the study start date (Day 1)
Total Number of Bleeds
Ramy czasowe: For the duration of 11 years before the study start date (Day 1)
For the duration of 11 years before the study start date (Day 1)
Number of Joint Bleed, Muscle Bleeds and Other Bleed Events in Participants
Ramy czasowe: For the duration of 11 years before the study start date (Day 1)
Joint bleeds included traumatic and spontaneous joint bleeds, muscle bleeds included traumatic and spontaneous soft tissue bleeds and other bleed events included intracranial bleed, gastrointestinal bleed and urinary tract bleed.
For the duration of 11 years before the study start date (Day 1)
Gilbert Joint Score
Ramy czasowe: Data analyzed on study start Day 1 for the duration of 4 years (2005 to 2009)
Gilbert joint score was an instrument to measure joint health in the domain of body structure and function (i.e. impairment), of the joints most commonly affected by bleeding in haemophilia - knees, ankles, elbows. Total score ranged from 0-100, evaluating ankle, knee and elbow, where "0" indicated normal joint function, "100" indicated worst joint function, where higher values indicated more impairment in joints.
Data analyzed on study start Day 1 for the duration of 4 years (2005 to 2009)
Haemophilia Joint Health Score
Ramy czasowe: Data analyzed on study start Day 1 for the duration of 6 years (2009 to 2015)
Haemophilia Joint Health Score (HJHS) was used to assess joint damage in participants with haemophilia. Total score ranges from 0 to 124, where, 0 indicates normal function, and 124 indicates worst joint function, higher values indicated more damage in joints.
Data analyzed on study start Day 1 for the duration of 6 years (2009 to 2015)
Percentage of Participants With Surgeries
Ramy czasowe: For the duration of 11 years before the study start date (Day 1)
Percentage of participants who had any type of surgery (arthrodesis, surgery on foot, nose, elbow, hand or shoulder, surgery on hip, surgery on knee, tooth extraction, venous port or any other) during the data observation period were reported.
For the duration of 11 years before the study start date (Day 1)
Average Relative Dose Intensity of Factor Concentrate Dispensed for Haemophilia A or B Participants on Prophylaxis Based on Dispensed Volume of Units
Ramy czasowe: For the duration of 11 years before the study start date (Day 1)
Relative dose intensity based on dispensed volume of units was calculated using annual dispensed volume of factor concentrate per participant divided by annual prescribed dose per participant. Data was analysed for 2 categories separately: children/adolescents and adults.
For the duration of 11 years before the study start date (Day 1)
Average Cost of Replacement Treatment (Benefix or Refacto/Refacto AF) Related to Bleed Event
Ramy czasowe: For the duration of 11 years before the study start date (Day 1)
For the duration of 11 years before the study start date (Day 1)

Miary wyników drugorzędnych

Miara wyniku
Ramy czasowe
Average Annual Cost of Prescribed Factor Concentrate and Dispensed Replacement Treatment
Ramy czasowe: For the duration of 11 years before the study start date (Day 1)
For the duration of 11 years before the study start date (Day 1)
Average Cost of Replacement Treatment Related to Invasive Procedures
Ramy czasowe: For the duration of 11 years before the study start date (Day 1)
For the duration of 11 years before the study start date (Day 1)

Inne miary wyników

Miara wyniku
Opis środka
Ramy czasowe
Average Annual Cost of Replacement Treatment Related to Invasive Procedures
Ramy czasowe: Data analyzed on study start Day 1 for Year 1 (2005) and Year 7 (2011)
Data analyzed on study start Day 1 for Year 1 (2005) and Year 7 (2011)
Average Cost for Replacement Treatment Related to Invasive Procedures
Ramy czasowe: Data analyzed on study start Day 1 for Year 1 (2005) and Year 7 (2011)
The average cost for replacement treatment related to invasive procedures was derived from the percentage of total costs for dispensed replacement therapy and calculated as: cost of factor concentrate used for invasive procedures/ total cost of factor concentrate.
Data analyzed on study start Day 1 for Year 1 (2005) and Year 7 (2011)

Współpracownicy i badacze

Tutaj znajdziesz osoby i organizacje zaangażowane w to badanie.

Sponsor

Pfizer

Publikacje i pomocne linki

Osoba odpowiedzialna za wprowadzenie informacji o badaniu dobrowolnie udostępnia te publikacje. Mogą one dotyczyć wszystkiego, co jest związane z badaniem.

Przydatne linki

Daty zapisu na studia

Daty te śledzą postęp w przesyłaniu rekordów badań i podsumowań wyników do ClinicalTrials.gov. Zapisy badań i zgłoszone wyniki są przeglądane przez National Library of Medicine (NLM), aby upewnić się, że spełniają określone standardy kontroli jakości, zanim zostaną opublikowane na publicznej stronie internetowej.

Główne daty studiów

Rozpoczęcie studiów (Rzeczywisty)

11 stycznia 2016

Zakończenie podstawowe (Rzeczywisty)

31 grudnia 2017

Ukończenie studiów (Rzeczywisty)

31 grudnia 2017

Daty rejestracji na studia

Pierwszy przesłany

16 grudnia 2015

Pierwszy przesłany, który spełnia kryteria kontroli jakości

11 kwietnia 2016

Pierwszy wysłany (Oszacować)

15 kwietnia 2016

Aktualizacje rekordów badań

Ostatnia wysłana aktualizacja (Rzeczywisty)

3 maja 2019

Ostatnia przesłana aktualizacja, która spełniała kryteria kontroli jakości

30 stycznia 2019

Ostatnia weryfikacja

1 stycznia 2019

Więcej informacji

Terminy związane z tym badaniem

Dodatkowe istotne warunki MeSH

Inne numery identyfikacyjne badania

  • B1821054

Plan dla danych uczestnika indywidualnego (IPD)

Planujesz udostępniać dane poszczególnych uczestników (IPD)?

NIE

Opis planu IPD

Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.

Te informacje zostały pobrane bezpośrednio ze strony internetowej clinicaltrials.gov bez żadnych zmian. Jeśli chcesz zmienić, usunąć lub zaktualizować dane swojego badania, skontaktuj się z register@clinicaltrials.gov. Gdy tylko zmiana zostanie wprowadzona na stronie clinicaltrials.gov, zostanie ona automatycznie zaktualizowana również na naszej stronie internetowej .

Badania kliniczne na Hemofilia A

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