Page last updated: 2024-11-06

prednisolone and Hemophilia B

prednisolone has been researched along with Hemophilia B in 9 studies

Prednisolone: A glucocorticoid with the general properties of the corticosteroids. It is the drug of choice for all conditions in which routine systemic corticosteroid therapy is indicated, except adrenal deficiency states.
prednisolone : A glucocorticoid that is prednisone in which the oxo group at position 11 has been reduced to the corresponding beta-hydroxy group. It is a drug metabolite of prednisone.

Hemophilia B: A deficiency of blood coagulation factor IX inherited as an X-linked disorder. (Also known as Christmas Disease, after the first patient studied in detail, not the holy day.) Historical and clinical features resemble those in classic hemophilia (HEMOPHILIA A), but patients present with fewer symptoms. Severity of bleeding is usually similar in members of a single family. Many patients are asymptomatic until the hemostatic system is stressed by surgery or trauma. Treatment is similar to that for hemophilia A. (From Cecil Textbook of Medicine, 19th ed, p1008)

Research Excerpts

ExcerptRelevanceReference
"A double-blind controlled study was carried out to investigate the effectiveness of treatment with factor VIII or factor IX concentrate and a reducing dose of prednisolone in contolling haematuria in patients with haemophilia and Christmas disease."9.04A comparison of coagulation factor replacement with and without prednisolone in the treatment of haematuria in haemophilia and Christmas disease. ( Kernoff, PB; Matthews, JM; McLennan, CR; Rainsford, SG; Rizza, CR, 1977)
"A 31-year-old man with mild hemophilia B developed a herniated disc treated with prednisolone for back pain."7.91Prednisolone treatment induced temporary factor IX normalization in mild hemophilia B who required an epidural infiltration: A case report. ( Barbay, V; Billoir, P; Carrette, M; Chamouni, P; Feugray, G; Fretigny, M; Le Cam Duchez, V, 2019)
"A double-blind controlled study was carried out to investigate the effectiveness of treatment with factor VIII or factor IX concentrate and a reducing dose of prednisolone in contolling haematuria in patients with haemophilia and Christmas disease."5.04A comparison of coagulation factor replacement with and without prednisolone in the treatment of haematuria in haemophilia and Christmas disease. ( Kernoff, PB; Matthews, JM; McLennan, CR; Rainsford, SG; Rizza, CR, 1977)
"A 31-year-old man with mild hemophilia B developed a herniated disc treated with prednisolone for back pain."3.91Prednisolone treatment induced temporary factor IX normalization in mild hemophilia B who required an epidural infiltration: A case report. ( Barbay, V; Billoir, P; Carrette, M; Chamouni, P; Feugray, G; Fretigny, M; Le Cam Duchez, V, 2019)
" He also had troublesome psoriasis for which he was receiving acitretin with UVB therapy and had recently discontinued low-dose daily prednisolone for associated arthropathy."3.69Multiple dermatofibromas in a patient with HIV infection. ( Armstrong, DK; Burrows, D; Irvine, A; Mayne, EE; Walsh, MY, 1995)
"The bleeding was effectively controlled within 1 to 48 h in five of nine bleeding episodes."2.69Controlling acute bleeding episodes with recombinant factor VIIa in haemophiliacs with inhibitor: continuous infusion and bolus injection. ( Angchaisuksiri, P; Chuansumrit, A; Hathirat, P; Isarangkura, P; Jorgensen, LN; Sriudomporn, N; Tanpowpong, K, 2000)

Research

Studies (9)

TimeframeStudies, this research(%)All Research%
pre-19904 (44.44)18.7374
1990's1 (11.11)18.2507
2000's1 (11.11)29.6817
2010's2 (22.22)24.3611
2020's1 (11.11)2.80

Authors

AuthorsStudies
Chandrakumara, J1
Wijesundara, M1
Amarakoon, G1
Miesbach, W1
Meijer, K1
Coppens, M1
Kampmann, P1
Klamroth, R1
Schutgens, R1
Tangelder, M1
Castaman, G1
Schwäble, J1
Bonig, H1
Seifried, E1
Cattaneo, F1
Meyer, C1
Leebeek, FWG1
Carrette, M1
Feugray, G1
Chamouni, P1
Le Cam Duchez, V1
Fretigny, M1
Barbay, V1
Billoir, P1
Egorova, LV1
Papaian, LP1
Khrolova, PV1
Volkova, SD1
Fedorova, ZD1
Armstrong, DK1
Irvine, A1
Walsh, MY1
Mayne, EE1
Burrows, D1
Chuansumrit, A1
Isarangkura, P1
Angchaisuksiri, P1
Sriudomporn, N1
Tanpowpong, K1
Hathirat, P1
Jorgensen, LN1
Rizza, CR1
Kernoff, PB1
Matthews, JM1
McLennan, CR1
Rainsford, SG1
Schimpf, K1
Handley, DA1
Lawrence, JR1

Clinical Trials (2)

Trial Overview

TrialPhaseEnrollmentStudy TypeStart DateStatus
A Phase I/II, Open-label, Uncontrolled, Single-dose, Dose-ascending, Multi-centre Trial Investigating an Adeno-associated Viral Vector Containing a Codon-optimized Human Factor IX Gene (AAV5-hFIX) Administered to Adult Patients With Severe or Moderately S[NCT02396342]Phase 1/Phase 210 participants (Actual)Interventional2015-06-10Completed
A Clinical Study Evaluating the Safety, Tolerability, and Initial Efficacy of Single Intravenous Infusion of JWK007 in Patients With Duchenne Muscular Dystrophy (DMD)[NCT06114056]Phase 16 participants (Anticipated)Interventional2023-11-10Recruiting
[information is prepared from clinicaltrials.gov, extracted Sep-2024]

Trial Outcomes

Number of Participants With Adverse Events

(NCT02396342)
Timeframe: From AMT-060 infusion through end of study (5 years post-dose)

Interventionparticipants (Number)
AAV5-hFIX Low Dose (Cohort 1)5
AAV5-hFIX High Dose (Cohort 2)5

Number of Subjects Developing Neutralizing Antibodies to AAV5

(NCT02396342)
Timeframe: From AMT-060 infusion through end of study (5 years post dose)

Interventionparticipants (Number)
AAV5-hFIX Low Dose (Cohort 1)5
AAV5-hFIX High Dose (Cohort 2)5

Number of Subjects With a Positive AAV5 Capsid-specific T Cell Response

Specific AAV5 response (results >17 SFC/million PBMCs) were regarded as positive. (NCT02396342)
Timeframe: From AMT-060 infusion through 26 weeks post-dose

Interventionparticipants (Number)
AAV5-hFIX Low Dose (Cohort 1)1
AAV5-hFIX High Dose (Cohort 2)0

Number of Subjects With Antibodies to FIX

(NCT02396342)
Timeframe: From AMT-060 infusion through the end of study (5 years post dose)

Interventionparticipants (Number)
AAV5-hFIX Low Dose (Cohort 1)1
AAV5-hFIX High Dose (Cohort 2)0

Number of Subjects With Clinically Significant Inflammatory Markers: IL-1β, IL-2, IL-6, INFγ, MCP-1

(NCT02396342)
Timeframe: From AMT-060 infusion through 18 weeks post dose

Interventionparticipants (Number)
AAV5-hFIX Low Dose (Cohort 1)0
AAV5-hFIX High Dose (Cohort 2)0

Number of Subjects With FIX Inhibitors

(NCT02396342)
Timeframe: From AMT-060 infusion through the end of study (5 years post dose)

Interventionparticipants (Number)
AAV5-hFIX Low Dose (Cohort 1)0
AAV5-hFIX High Dose (Cohort 2)0

Change From Baseline in Short Form-36 (SF-36) Quality of Life (QoL) Scores

Scores range from 0 to 100, with a higher score defining a more favorable health state. (NCT02396342)
Timeframe: From AMT-060 infusion through the end of study (5 years post dose)

,
Interventionscore on a scale (Mean)
Physical FunctioningRole-PhysicalBodily PainGeneral HealthVitalitySocial FunctioningRole-EmotionalMental Health
AAV5-hFIX High Dose (Cohort 2)-7.00-10.001.20-2.40-6.25-5.00-10.00-9.00
AAV5-hFIX Low Dose (Cohort 1)0.00-15.00-9.00-0.80-11.25-20.00-13.33-13.00

FIX-replacement-therapy-free FIX Activity

FIX activity measured any time from 72 hours after latest FIX replacement therapy administration and until next administration of FIX replacement therapy. Only assessments performed more than 10 days after most recent FIX-replacement therapy administration included. (NCT02396342)
Timeframe: From AMT-060 infusion through end of study (5 years post-dose)

,
InterventionPercent FIX activity (Mean)
one-stage aPTT assayamidolytic/chromogenic assay
AAV5-hFIX High Dose (Cohort 2)6.604.74
AAV5-hFIX Low Dose (Cohort 1)7.434.58

Time to Vector DNA Stopped Shedding From Blood, Nasal Secretions, Saliva, Urine, Feces, and Semen

(NCT02396342)
Timeframe: From AMT-060 infusion through end of study (5 years post dose).

,
InterventionDays (Mean)
BloodNasal secretionsSalivaUrineFecesSemen
AAV5-hFIX High Dose (Cohort 2)705.4108.4129.282.0165.0157.2
AAV5-hFIX Low Dose (Cohort 1)508.883.475.846.474.0227.8

Total Annualized Bleeding Rate (ABR)

Annualized: Sum of post-treatment bleeding episodes divided by subjects' average number of years (365.25 days) from treatment start to until the data cutoff date. (NCT02396342)
Timeframe: From AMT-060 infusion through end of study (5 years post-dose)

,
Interventionbleeds/year/subject (Mean)
One Year Prior to ScreeningPost-tapering Period
AAV5-hFIX High Dose (Cohort 2)4.000.71
AAV5-hFIX Low Dose (Cohort 1)14.405.39

Total Consumption of FIX Replacement Therapy

(NCT02396342)
Timeframe: From AMT-060 infusion through end of study (5 years post dose).

,
InterventionIU (Mean)
One year prior to screeningPost-tapering period
AAV5-hFIX High Dose (Cohort 2)23377885800
AAV5-hFIX Low Dose (Cohort 1)326532252950

Total IgG and IgM Antibody Titers to AAV5

For subjects with a titer of 109350 and 50, the actual titer is >109350 and <50. (NCT02396342)
Timeframe: AMT-060 infusion through end of study (5 years post dose)

,
InterventionTiter (Number)
IgG (subject 1)IgG (subject 2)IgG (subject 3)IgG (subject 4)IgG (subject 5)IgM (subject 1)IgM (subject 2)IgM (subject 3)IgM (subject 4)IgM (subject 5)
AAV5-hFIX High Dose (Cohort 2)109350109350109350107344109350300712000066495050
AAV5-hFIX Low Dose (Cohort 1)7949910935010935010935010935056132155711568809

Trials

3 trials available for prednisolone and Hemophilia B

ArticleYear
Gene therapy with adeno-associated virus vector 5-human factor IX in adults with hemophilia B.
    Blood, 2018, 03-01, Volume: 131, Issue:9

    Topics: Adult; Dependovirus; Factor IX; Female; Genetic Therapy; Genetic Vectors; Hemophilia B; Humans; Male

2018
Gene therapy with adeno-associated virus vector 5-human factor IX in adults with hemophilia B.
    Blood, 2018, 03-01, Volume: 131, Issue:9

    Topics: Adult; Dependovirus; Factor IX; Female; Genetic Therapy; Genetic Vectors; Hemophilia B; Humans; Male

2018
Gene therapy with adeno-associated virus vector 5-human factor IX in adults with hemophilia B.
    Blood, 2018, 03-01, Volume: 131, Issue:9

    Topics: Adult; Dependovirus; Factor IX; Female; Genetic Therapy; Genetic Vectors; Hemophilia B; Humans; Male

2018
Gene therapy with adeno-associated virus vector 5-human factor IX in adults with hemophilia B.
    Blood, 2018, 03-01, Volume: 131, Issue:9

    Topics: Adult; Dependovirus; Factor IX; Female; Genetic Therapy; Genetic Vectors; Hemophilia B; Humans; Male

2018
Controlling acute bleeding episodes with recombinant factor VIIa in haemophiliacs with inhibitor: continuous infusion and bolus injection.
    Haemophilia : the official journal of the World Federation of Hemophilia, 2000, Volume: 6, Issue:2

    Topics: Adult; Anti-Inflammatory Agents; Antifibrinolytic Agents; Child, Preschool; Disease Management; Fact

2000
A comparison of coagulation factor replacement with and without prednisolone in the treatment of haematuria in haemophilia and Christmas disease.
    Thrombosis and haemostasis, 1977, Feb-28, Volume: 37, Issue:1

    Topics: Clinical Trials as Topic; Drug Therapy, Combination; Factor IX; Factor VIII; Hematuria; Hemophilia A

1977

Other Studies

6 other studies available for prednisolone and Hemophilia B

ArticleYear
A child diagnosed with severe hemophilia A presenting with nephrotic syndrome: a case report.
    Journal of medical case reports, 2023, May-20, Volume: 17, Issue:1

    Topics: Child; Factor VIII; Hemophilia A; Hemophilia B; Humans; Male; Nephrotic Syndrome; Prednisolone

2023
Prednisolone treatment induced temporary factor IX normalization in mild hemophilia B who required an epidural infiltration: A case report.
    Transfusion and apheresis science : official journal of the World Apheresis Association : official journal of the European Society for Haemapheresis, 2019, Volume: 58, Issue:4

    Topics: Adult; Factor IX; Hemophilia B; Humans; Intervertebral Disc Displacement; Male; Partial Thromboplast

2019
[Hormone therapy of renal hemorrhages in hemophiliacs].
    Sovetskaia meditsina, 1982, Issue:8

    Topics: Adolescent; Adrenal Cortex Hormones; Adult; Hematuria; Hemophilia A; Hemophilia B; Hemorrhage; Human

1982
Multiple dermatofibromas in a patient with HIV infection.
    Clinical and experimental dermatology, 1995, Volume: 20, Issue:6

    Topics: Acitretin; Adult; Hemophilia B; Histiocytoma, Benign Fibrous; HIV Infections; Humans; Male; Predniso

1995
[Substitution treatment in hemophilia. Critical measures and complications].
    Wiener medizinische Wochenschrift (1946), 1977, Jun-10, Volume: 127, Issue:11

    Topics: Blood Transfusion; Disseminated Intravascular Coagulation; Factor IX; Factor VIII; Half-Life; Hemoph

1977
Factor-IX deficiency in the nephrotic syndrome.
    Lancet (London, England), 1967, May-20, Volume: 1, Issue:7499

    Topics: Adolescent; Biological Assay; Blood Coagulation Tests; Child; Hemophilia B; Humans; Male; Nephrotic

1967