FrancophonIA/ELRC3.0 huggingface dataset:
English: The MAH shall plan to include regular progress reports of the registry in the PSUR and provide interim study reports every 2 years until the registry finishes.
Danish: Indehaveren af markedsføringstilladelsen skal inkludere regelmæssige rapporter af fremdriften af registret i PSUR'en og skal udarbejde interime studierapporter hvert andet år, indtil registret er færdigt.
English: The efficacy of Strimvelis was evaluated in a 3-year open-label, prospective study in children who lacked a sibling HLA matched stem cell donor and were either failing to respond adequately to PEG-ADA, were intolerant or did not have access to it.
Danish: Effekten af Strimvelis blev vurderet i et 3-årigt åbent, prospektivt studie med børn, som ikke havde en søskende, som kunne være HLA-matchende stamcelledonor, og som enten ikke responderede tilstrækkeligt på PEG-ADA, var intolerant over for det, eller som ikke havde adgang til det.
English: The ones marked * may be related to busulfan.
Danish: De bivirkninger, der er markeret med *, kan skyldes behandlingen med busulfan.
English: • if there was a delay in the medicine reaching the clinic where treatment is being carried out.
Danish: • hvis der skete en forsinkelse under transporten af medicinen til det hospital, hvor behandlingen skulle udføres.
English: There were no adverse effects on survival, haematological parameters or histopathology of major organs, apart from body weight loss and atrophy in the testes and ovaries consistent with administration of busulfan.
Danish: Der sås ingen uønskede virkninger på overlevelse, hæmatologiske parametre eller histopatologi for større organer bortset fra vægttab og atrofi i testikler og ovarier forenelig med administration af busulfan.
English: Interventionfree survival in this pivotal population (defined as survival without the requirement for long-term (≥3 month) re-introduction of PEG-ADA, or stem cell transplant) was 92% (11/12 subjects) (82% (14/17 subjects) for integrated population).
Danish: Interventionsfri overlevelse i denne pivotale population (defineret som overlevelse uden behov for langvarig (≥ 3 måneder) genoptagelse af PEG-ADA eller stamcelletransplantation) var 92 % (11/12 forsøgspersoner) (82 % (14/17 forsøgspersoner) for den integrerede population).
English: The quantitative information regarding CD34+ cells/kg and total cells in the product is presented in the labelling for each batch.
Danish: De kvantitative oplysninger vedrørende CD34+-celler/kg og det totale antal celler i præparatet er anført på etiketten for hver batch.
English: All the identified adverse reactions in the table (apart from those potentially related to busulfan) are considered to be related to immune reconstitution, due to their nature and timing.
Danish: Alle de anførte bivirkninger i tabellen (bortset fra dem, der muligvis er relateret til busulfan) anses, på grund af deres art og tidsmæssige sammenhæng, for at være forbundet med immunrekonstitution.
English: Strimvelis is for intravenous infusion.
Danish: Strimvelis er til intravenøs infusion.
English: At least 3 weeks before treatment
Danish: Mindst 3 uger før behandlingen
English: BATCH NUMBER, DONATION AND PRODUCT CODES Lot:
Danish: FREMSTILLERENS BATCHNUMMER, DONATIONS- OG PRODUKTKODER Lot:
English: Strimvelis is transported directly to the medical facility where the infusion will be administered.
Danish: Strimvelis transporteres direkte til det hospital, hvor infusionen skal gives.
English: If the product contains less than 2 million CD34+ cells/kg, the treating physician should make a decision whether to proceed with administration, based on an individual benefit risk assessment.
Danish: Hvis præparatet indeholder under 2 millioner CD34+-celler/kg, skal den behandlende læge beslutte, om administrationen skal gennemføres ud fra en individuel vurdering af benefit/risk-forholdet.
English: Stage two quality control results will only be available after the product has been infused.
Danish: Resultaterne for den anden kvalitetskontrol vil først foreligge, efter præparatet er blevet infunderet.
English: Another subject needed transient PEG-ADA administration due to an autoimmune event (see section 4.4).
Danish: En anden forsøgsperson havde behov for forbigående PEG-ADA-administration grundet en autoimmun hændelse (se pkt.
English: Reproductive and developmental studies have not been conducted.
Danish: Der er ikke udført reproduktions- og udviklingsstudier.
English: About 15 to 30 minutes before treatment
Danish: Ca. 15-30 minutter før behandlingen
English: by a drip into a vein.
Danish: i et drop i en vene.
English: Very common > 1/10 Common ≥1/100 to <1/10
Danish: ≥ 1/100 til < 1/10
English: b Based on the 3 year post-treatment period for survival and severe infections, and the data collected at the 3 year visit for T-lymphocytes and dAXP.
Danish: b Baseret på den 3-årige periode efter behandlingen for overlevelse og svære infektioner og data indsamlet ved 3-årsbesøget for T-lymfocytter og dAXP.
English: Strimvelis is intended solely for autologous use and should never be administered to any patient other than the original CD34+ cell donor.
Danish: Strimvelis er udelukkende beregnet til autolog brug og må aldrig gives til andre patienter end den oprindelige CD34+-celledonor.
English: Unused medicine must be disposed of in compliance with the local biosafety guidelines.
Danish: Ikke anvendt lægemiddel skal bortskaffes i overensstemmelse med de lokale retningslinjer for biosikkerhed.
English: Read all of this leaflet carefully before you (or your child) are given this medicine because it contains important information for you.
Danish: Læs denne indlægsseddel grundigt, inden du (eller dit barn, hvis han/hun er patienten) får dette lægemiddel, da den indeholder vigtige oplysninger.
English: to make the bone marrow ready for Strimvelis
Danish: for at forberede knoglemarven på Strimvelis
English: No data from clinical studies are available regarding overdose of Strimvelis.
Danish: Der foreligger ingen data fra kliniske studier om overdosering af Strimvelis.
English: • blood test results positive for antineutrophil cytoplasmic antibody and smooth muscle antibody
Danish: • positive blodprøveresultater for anti-neutrofilocyt cytoplasma-antistof og glat muskel-antistof
English: These autoimmune adverse reactions were reported for subjects post-gene therapy.
Danish: Disse autoimmune bivirkninger blev rapporteret hos forsøgspersoner efter genterapi.
English: Post treatment
Danish: Efter behandling
English: All patients received busulfan conditioning prior to gene therapy, with most receiving a total dose of 4 mg/kg intravenously over 2 consecutive days prior to CD34+ infusion.
Danish: Alle patienter fik konditionering med busulfan inden genterapien, og de fleste fik en samlet dosis på 4 mg/kg intravenøst over 2 på hinanden følgende dage inden infusion af CD34+.
English: No immunogenicity testing has been conducted with Strimvelis.
Danish: Der er ikke udført immungenicitetstestning af Strimvelis.
English: 3 days and 2 days before treatment
Danish: 3 dage og 2 dage før behandlingen
English: Strimvelis is used to treat ADA-SCID when there is no suitable match from a family member to donate stem cells from their bone marrow for a transplant.
Danish: Strimvelis bruges til at behandle ADA-SCID, når der ikke blandt familiemedlemmerne er en person, der er egnet til at donere stamceller fra knoglemarven til en transplantation.
English: It is unknown whether Strimvelis is excreted in human milk.
Danish: Det vides ikke, om Strimvelis udskilles i human mælk.
English: This medicine contains approximately 3.5 mg sodium per millilitre.
Danish: Dette lægemiddel indeholder ca. 3,5 mg natrium pr. milliliter.
English: CD34+ cells/kg:
Danish: CD34+-celler/kg:
English: Patients who have previously tested positive for hepatitis C can be treated with Strimvelis, provided they demonstrate absence of ongoing infection using a nucleic acid test with a limit of quantification of ≤15 international units/ml.
Danish: Patienter, der tidligere er testet positive for hepatitis C, kan behandles med Strimvelis, hvis en nukleinsyreamplifikationstest med kvantifikationsgrænse på ≤ 15 internationale enheder/ml viser fravær af igangværende infektion.
English: Strimvelis is an autologous cellular therapy.
Danish: Strimvelis er autolog celleterapi.
English: MINIMUM PARTICULARS TO APPEAR ON SMALL IMMEDIATE PACKAGING UNITS INFUSION BAG
Danish: MINDSTEKRAV TIL MÆRKNING PÅ SMÅ INDRE EMBALLAGER INFUSIONSPOSE
English: As Strimvelis will be administered following busulfan conditioning, patients of childbearing potential must use reliable barrier contraception during administration of Strimvelis and for at least 6 months afterwards.
Danish: Da Strimvelis administreres efter konditionering med busulfan, skal fertile patienter anvende sikker barrierekontraception, når Strimvelis administreres og i mindst 6 måneder derefter.
English: Treatment sample of stem cells collected to make Strimvelis (see above)
Danish: Behandlingsprøve af stamceller udtages til fremstilling af Strimvelis (se ovenfor)
English: Strimvelis is used to treat a serious condition called ADA-SCID (Adenosine Deaminase-Severe Combined Immune Deficiency).
Danish: Strimvelis bruges til at behandle en alvorlig sygdom kaldet ADA-SCID (svær kombineret adenosindeaminase-immundefekt).
English: It works by putting a new gene into stem cells in the bone marrow so they can make ADA.
Danish: Det virker ved at indføre et nyt gen i stamcellerne i knoglemarven, så de kan producere ADA.
English: Successful generation of T cells after Strimvelis is also likely to be affected by residual thymic function which can become impaired in older children.
Danish: Succesfuld dannelse af T-celler efter behandling med Strimvelis kan også blive påvirket af tilbageværende thymusfunktion, som kan blive nedsat hos ældre børn.
English: It can only be given in a specialised hospital, and by a doctor who is experienced in treating patients with ADA-SCID and in using this type of medicine.
Danish: Det må kun indgives på et specialiseret hospital af en læge, som har erfaring med behandling af patienter med ACA-SCID og med brug af denne type medicin.
English: Four subjects had previously received an unsuccessful stem cell transplant from a haploidentical donor and 15 of 18 subjects had previously received prior enzyme replacement therapy with polyethylene-glycol-modified bovine adenosine deaminase (PEG-ADA).
Danish: Fire forsøgspersoner havde tidligere gennemgået en mislykket transplantation med stamceller fra en haploidentisk donor, og 15 ud af 18 forsøgspersoner havde tidligere fået enzymsubstitutionsbehandling med PEG-ADA (polyethylenglycolmodificeret bovin adenosindeaminase).
English: Local biosafety guidelines applicable should be followed.
Danish: Lokale retningslinjer for biosikkerhed skal følges.
English: Strimvelis is made specially for each patient, using the patient's own bone marrow cells.
Danish: Strimvelis fremstilles specielt til hver enkelt patient, idet patientens egne knoglemarvsceller anvendes.
English: However, vector insertions into chromosomal regions previously associated with leukaemia in comparable trials of gene therapy in Wiskott Aldrich Syndrome, X-SCID and Chronic Granulomatous Disease have been documented.
Danish: Der er dog dokumenteret vektorindsættelse i kromosomale regioner, der tidligere er forbundet med leukæmi i sammenlignelige studier med genterapi til Wiskott Aldrichs syndrom, X-SCID og kronisk granulomatøs sygdom.
English: Treatment failure was observed in a patient treated in the clinical trials with <2 millions CD34+cell/kg.
Danish: Der blev observeret behandlingssvigt hos en patient, som i de kliniske studier blev behandlet med < 2 millioner CD34+-celler/kg.
English: Strimvelis is a type of medicine called a gene therapy.
Danish: Strimvelis er en type lægemiddel, som bruges til genterapi.
English: An antihistamine medicine may be given to make it less likely that you will react to the infusion
Danish: Der kan gives et antihistaminpræparat for at mindske risikoen for, at du får bivirkninger i forbindelse med infusionen
English: No cases of leukaemia or myelodysplasia have been reported following treatment with Strimvelis.
Danish: Der er ikke rapporteret om leukæmi eller myelodysplasi efter behandling med Strimvelis.
English: The concentration is 1-10 million CD34+ cells/ml.
Danish: Koncentrationen er 1-10 millioner CD34+-celler/ml.
English: • runny or blocked nose (allergic rhinitis)
Danish: • løbende eller tilstoppet næse (allergisk rhinitis)
English: People with ADA-SCID cannot produce enough of an enzyme called adenosine deaminase (ADA) because the gene to make it is faulty.
Danish: Personer med ADA-SCID kan ikke producere nok af et enzym, som kaldes adenosindeaminase (ADA), fordi det gen, som koder for ADA, ikke virker, som det skal.
English: The patient must be able to donate adequate CD34+ cells to deliver the minimum 4 million purified CD34+ cells/kg, required for manufacture of Strimvelis.
Danish: Patienten skal kunne donere en tilstrækkelig mængde CD34+-celler til at opnå de mindst 4 millioner rensede CD34+-celler/kg, som er nødvendige for at kunne fremstille Strimvelis.
English: Adverse events related to the use of central venous catheters (CVCs) have been reported (e.g., serious CVC infections and thrombosis in the device).
Danish: Der er rapporteret om uønskede hændelser i forbindelse med brug af centrale venekatetre (CVC) (f.eks. alvorlige CVC-infektioner og trombose i devicet).
English: • wheezing, difficulty breathing (asthma)
Danish: • hvæsende vejrtrækning, vejrtrækningsbesvær (astma)
English: Strimvelis is intended solely for autologous use.
Danish: Strimvelis er alene beregnet til autolog brug.
English: Some patients have had to resume long-term enzyme replacement therapy and/or receive a stem cell transplant (see section 5.1).
Danish: For nogle patienter har det været nødvendigt at genoptage langvarig enzymsubstitutionsbehandling og/eller undergå en knoglemarvstransplantation (se pkt.
English: A total of 18 patients with ADA-SCID were treated with Strimvelis as part of one open-label pivotal trial (AD1115611; N=12), two early open-label pilot studies (AD1117054/AD1117056; N=3), and a compassionate use program (AD1117064; N=3).
Danish: I alt 18 patienter med ADA-SCID blev behandlet med Strimvelis i et åbent pivotalstudie (AD1115611; N=12), i to tidlige åbne pilotstudier (AD1117054/AD1117056; N=3) og i et compassionate use program (AD1117064; N=3).
English: The educational materials should address the following safety concerns/key elements: Autoimmunity, Unsuccessful response to gene therapy, and Malignancy due to insertional oncogenesis (e.g. leukaemia, myelodysplasia).
Danish: Uddannelsesmaterialet skal omhandle følgende sikkerhedsanliggender/hovedelementer: Autoimmunitet, manglende respons på genterapi, og malignitet pga. insertionel mutagenese (f.eks. leukæmi, myelodysplasi).
English: B cell function: All 12 subjects treated in the pivotal study were receiving IVIG therapy at the time of screening, and 7 subjects (58%) had discontinued IVIG use during 0-3 years follow-up after gene therapy.
Danish: B-cellefunktion: Alle 12 forsøgspersoner, som blev behandlet i pivotalstudiet, fik behandling med intravenøst immunglobulin (IVIG) på screeningstidspunktet, og 7 forsøgspersoner (58 %) var stoppet med IVIG i løbet af opfølgningsperioden 0-3 år efter genterapi.
English: One subject treated in a pilot study did not have PEG-ADA re-introduction data, and thus was excluded from the intervention-free survival in the integrated population.
Danish: Der manglede data for genoptagelse af PEG-ADA for en forsøgsperson, som blev behandlet i et pilotstudie, og som derfor ikke blev inkluderet som interventionsfri overlevelse i den integrerede population.
English: Strimvelis should be used with caution in patients with hypersensitivity to aminoglycosides or bovine serum albumin.
Danish: Der skal udvises forsigtighed ved brug af Strimvelis til patienter med overfølsomhed over for aminoglykosider eller bovint serumalbumin.
English: The median percentages of genetically modified cells in peripheral blood at one year and 3 years after treatment were 28% (range 6%-92%) and 30% (range 8%-101%) of CD19+, and 73% (range 20%-100%) and 67% (range 39%-82%) of CD3+ cells, respectively.
Danish: De mediane procenter af genmodificerede celler i perifert blod 1 og 3 år efter behandlingen var henholdsvis 28 % (6-92 %) og 30 % (8-101 %) for CD19+-celler og 73 % (20-100 %) og 67 % (39-82 %) for CD3+celler.
English: Name and address of the manufacturer responsible for batch release MolMed SpA
Danish: Navn og adresse på den fremstiller, der er ansvarlig for batchfrigivelse MolMed SpA
English: - are allergic to any of the ingredients of this medicine (listed in section 6) - have or have had a type of cancer called leukaemia or myelodysplasia - have tested positive for HIV or some other infections (your doctor will advise you about this) - have already been treated with gene therapy
Danish: Du (eller dit barn) må ikke få Strimvelis: - hvis du er allergisk over for nogen af indholdsstofferne (angivet i punkt 6) - hvis du har eller har haft myelodysplasi eller en type cancer, som kaldes leukæmi - hvis du er testet positiv for hiv eller andre infektioner (din læge vil rådgive dig om dette) - hvis du allerede er blevet behandlet med genterapi.
English: No. of severe infections
Danish: Antal svære infektioner
English: T-lymphocyte (CD3+) and NK (CD56+) cell counts improved following treatment with Strimvelis.
Danish: Antallet af T-lymfocytter (CD3+) og NK-celler (CD56+) steg efter behandlingen med Strimvelis.
English: Effects on male and female fertility have not been evaluated in animal studies.
Danish: Virkningerne på han- og hundyrs fertilitet er ikke blevet undersøgt i dyrestudier.
English: Cases of skin papillomas, abnormal serum protein electrophoresis and one case each of lipofibroma, pulmonary mass and decreased T-cell V beta repertoire were reported.
Danish: Der blev rapporteret om hudpapillom, abnorm serumproteinelektroforese og ét tilfælde af henholdsvis lipofibrom, pulmonal udfyldning og reduceret T-cellereceptor V-beta-repertoire.
English: • inflamed liver (autoimmune hepatitis)
Danish: • leverbetændelse (autoimmun hepatitis)
English: - Your doctor will give you a Patient Alert Card. Read it carefully and follow the instructions on it. - Always show the Patient Alert Card to your doctor or nurse when you see them or if you go to hospital.
Danish: - Vis altid patientoplysningskortet til din læge eller sundhedspersonalet, når du ser dem, eller hvis du skal på hospitalet.
English: Gently agitate the infusion bag to re-disperse any cellular aggregates, administer using a transfusion administration set with filter to remove any remaining cellular aggregates.
Danish: Vend forsigtigt infusionsposen op og ned for at dispergere eventuelle celleaggregater og udfør infusionen med et transfusionssæt med filter for at fjerne eventuelle tilbageværende celleaggregater.
English: Anaemiaa Neutropeniaa
Danish: Anæmia Neutropenia
English: The stem cell back-up is collected for use as rescue treatment should there be a failure during product manufacture, transplant failure, or prolonged bone marrow aplasia after treatment with Strimvelis.
Danish: Stamcellerreserven udtages i tilfælde af behov for nødbehandling, hvis der skulle opstå fejl under fremstillingen af præparatet, transplantationssvigt eller langvarig knoglemarvsaplasi efter behandlingen med Strimvelis.
English: Rate of severe infections per person year * Excluding data from one patient collected from the time of intervention with PEG-ADA (>3 months treatment) or hematopoietic stem cell transplantation.
Danish: Forekomst af svære infektioner pr. patientår * Bortset fra data fra én patient indsamlet fra interventionstidspunktet med PEG-ADA (>3 måneders behandling) eller hæmatopoietisk stamcelletransplantation
English: 67% (12 of 18) of Strimvelis treated patients had either autoimmune antibodies or other manifestations (e.g. autoimmune thrombocytopenia, autoimmune aplastic anaemia, autoimmune hepatitis and Guillain-Barré syndrome) (see section 4.8).
Danish: 67 % (12 af 18) af de patienter, som fik Strimvelis, havde enten autoimmune antistoffer eller andre manifestationer (f.eks. autoimmun trombocytopeni, autoimmun aplastisk anæmi, autoimmun hepatitis og Guillain-Barré-syndrom (se pkt.
English: Non-interventional PASS: In order to investigate the long term safety and efficacy of Strimvelis gene therapy, the MAH should conduct and submit the results of a long term prospective, non-interventional follow up study using data from a registry of patients with adenosine deaminase severe combined immunodeficiency (ADA-SCID) treated with Strimvelis.
Danish: Ikke-interventionssikkerhedsundersøgelser efter tilladelse til markedsføring (PASS): For at undersøge langtidssikkerheden og -virkningen af Strimvelisgenterapi skal indehaveren af markedsføringstilladelsen udføre et langtids-, prospektivt, ikke-interventions-opfølgningsstudie med data fra et register over patienter med svær kombineret adenosindeaminase-immundefekt (ADA-SCID) behandlet med Strimvelis samt indsende resultaterne.
English: In some cases, it might not be possible to go ahead with the planned treatment with Strimvelis.
Danish: Når behandlingen med Strimvelis ikke kan gennemføres I visse tilfælde vil det muligvis ikke kunne lade sig gøre at påbegynde den planlagte behandling med Strimvelis.
English: Strimvelis is made specially from the patient's own cells.
Danish: Strimvelis er fremstillet ud fra patientens egne celler.
English: Any unused medicinal product or waste material should be disposed of in accordance with local biosafety requirements.
Danish: Ikke anvendt lægemiddel samt affald heraf skal bortskaffes i henhold til lokale retningslinjer for biosikkerhed.
English: CD34+ cells derived from healthy human umbilical cord blood, transduced with the vector used for the production of Strimvelis, were administered intravenously to busulfan-conditioned mice.
Danish: CD34+-celler fra navlestrengsblod fra en rask person transduceret med den vektor, som bruges til fremstilling af Strimvelis, blev administreret intravenøst til mus efter konditionering med busulfan.
English: Findings that TREC (T cell receptor excision circles) in peripheral blood lymphocytes were increased above baseline (median 141, range 56 to 1542 copies/100ng DNA) at Years 1 and maintained to 3 post-treatment and that all subjects had evidence of polyclonal V-beta chains at one or more time points following gene therapy provides further supportive evidence of functional T cell development.
Danish: Resultater, der viser, at TREC (T-cellereceptor excision cirkler) i lymfocytter i perifert blod steg til over baseline-niveau (median 141, interval 56 til 1.542 kopier/100 ng DNA) efter 1 år og blev opretholdt 3 år efter behandlingen, og at alle forsøgspersoner viste tegn på polyklonale V-betakæder på et eller flere tidspunkter efter genterapi, giver yderligere understøttende dokumentation for udvikling af funktionelle T-celler.
English: A CD34+ stem cell back-up containing at least 1 million CD34+ cells per kg is required.
Danish: En reserve af CD34+-stamceller med mindst 1 million CD34+-celler pr. kg er nødvendig.
English: Treatment with Strimvelis resulted in a 100% survival rate at 3 years post therapy, a decrease in the severe infection rate, an increase in T-lymphocytes (CD3+) and all subjects having post-baseline venous red blood cell deoxyadenosine nucleotide (RBC dAXP) levels below pathological levels (>100 nmol/ml).
Danish: Behandling med Strimvelis gav en 100 % overlevelsesrate 3 år efter behandlingen, et fald i hyppigheden af svære infektioner, en stigning i T-lymfocytter (CD3+), og alle forsøgspersoner havde venøst RBC-dAXP (erytrocytdeoxyadenosinnukleotid)-niveau under patologisk niveau (> 100 nmol/ml) efter baseline.
English: Hypothyroidism Autoimmune thyroiditis
Danish: Autoimmun thyroiditis