ASH Clinical News ACN_4.13_full issue_Web | Page 44

Literature Scan Higher Childhood Leukemia Risk in Offspring Raises Questions About Hormonal Contraceptive Safety Children born to women who were using hormonal contraception within three months of pregnancy and during pregnancy have a higher risk of devel- oping myeloid leukemia than those born to mothers who were not using hormonal contraception, according to a nationwide cohort study pub- lished in Lancet Oncology. Results of the study suggest that exposure to hormonal contraception in utero is a potential predictor for child- hood leukemia – a disease with few established risk factors, given the known association between expo- sure to exogenous sex hormones and tumor development. “The strength of the associa- tions and the biologic plausibility of hormones causing cancer in children show that these results are strong enough to cause concern,” study author Marie Hargreave, PhD, from the Danish Cancer So- ciety Research Center in Copenha- gen, told ASH Clinical News. “Our results can have direct implications for clinical guidelines on how we use hormonal contraception up to pregnancy in the near future and will potentially protect future gen- erations of children from potential harmful exposures before birth.” Using data from the Danish Cancer Registry and the Danish Medical Birth Registry, investiga- tors identified 1,185,157 children born between 1996 and 2014. The researchers then used the Danish National Prescription Registry to determine children’s exposure to hormonal contraceptives, using the following definitions: • no use: mothers who never used contraception prior to birth (n=270,291) • previous use (defined as prescriptions filled): mothers who used hormonal contraceptives >3 months prior to the start of pregnancy (n=778,844) • recent use: mothers who used hormonal contraceptives ≤3 months prior to and during pregnancy (n=136,022) During a median of 9.3 years (range = 4.6-14.2 years) from birth, 606 children were diagnosed with either lymphoid leukemia (n=465) or myeloid leukemia (n=141). 42 ASH Clinical News Compared with children of women who reported never using hormonal contraception, children born to women with recent use had a significantly higher risk for any leukemia (primary endpoint; hazard ratio [HR] = 1.46; 95% CI 1.09- 1.96; p=0.011). Among children who were exposed to hormonal contraception in utero, the HR rose non-significantly to 1.78 (95% CI 0.95-3.31; p=0.070). JIVI ® [antihemophilic factor (recombinant), PEGylated-aucl] lyophilized powder for solution, for intravenous use Initial U.S. Approval: 2018 BRIEF SUMMARY CONSULT PACKAGE INSERT FOR FULL PRESCRIBING INFORMATION 1 INDICATIONS AND USAGE Jivi, antihemophilic factor (recombinant), PEGylated-aucl, is a recombinant DNA-derived, Factor VIII concentrate indicated for use in previously treated adults and adolescents (12 years of age and older) with hemophilia A (congenital Factor VIII deficiency) for: • On-demand treatment and control of bleeding episodes • Perioperative management of bleeding • Routine prophylaxis to reduce the frequency of bleeding episodes Limitations of Use Jivi is not indicated for use in children < 12 years of age due to greater risk for hypersensitivity reactions [see Use in Specific Populations (8.4)]. Jivi is not indicated for use in previously untreated patients (PUPs). Jivi is not indicated for the treatment of von Willebrand disease. 4 CONTRAINDICATIONS Jivi is contraindicated in patients who have a history of hypersensitivity reactions to the active substance, polyethylene glycol (PEG), mouse or hamster proteins, or other constituents of the product [see Description (11)]. There was no statistically signifi- cant association between previous use and lymphoid leukemia risk (HR=1.23; 95% CI 0.97-1.57; p=0.089) or between recent use and lymphoid leukemia risk 5.4 Monitoring Laboratory Tests • If monitoring of Factor VIII activity is performed, use a validated chromogenic assay or a selected validated one-stage clotting assay [see Dosage and Administration (2.1)]. • Laboratories intending to measure the Factor VIII activity of Jivi should check their procedures for accuracy. For Jivi, select silica-based one-stage assays may underestimate the Factor VIII activity of Jivi in plasma samples; some reagents, e.g., with kaolin-based activators, have the potential for overestimation 1 . Therefore, the suitability of the assay must be ascertained. If a validated one-stage clotting or chromogenic assay is not available locally, then use of a reference laboratory is recommended. • Monitor for development of Factor VIII inhibitors. Perform a Bethesda inhibitor assay if expected Factor VIII plasma levels are not attained or if bleeding is not controlled with the expected dose of Jivi. Use Bethesda Units (BU) to report inhibitor titers. 6 ADVERSE REACTIONS The most frequently (≥ 5%) reported adverse reactions in clinical trials in previously treated patients (PTPs) ≥ 12 years of age were headache, cough, nausea and fever (see Table 3). 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in clinical trials of another drug and may not reflect the rates observed in clinical practice. A total of 221 subjects constituted the safety population from three studies. Subjects who received Jivi for perioperative management (n=17) with treatment period of 2 to 3 weeks were excluded from pooled safety analysis but included in analysis for inhibitor development. The median EDs for adults and adolescents (≥ 12 years of age) was 131 EDs (range: 1–309) per subject; and the median EDs for subjects < 12 years of age was 53 EDs (range: 1–68) per subject. 5 WARNINGS AND PRECAUTIONS 5.1 Hypersensitivity Reactions Hypersensitivity reactions, including severe allergic reactions, have occurred with Jivi. Monitor patients for hypersensitivity symptoms. Early signs of hypersensitivity reactions, which can progress to anaphylaxis, may include chest or throat tightness, dizziness, mild hypotension and nausea. If hypersensitivity reactions occur, Table 3: Adverse Reactions reported for Jivi immediately discontinue administration and initiate appropriate MedDRA Standard All Subjects ≥12 treatment. System Organ Class Subjects years of age Jivi may contain trace amounts of mouse and hamster proteins Preferred term n (%) n (%) [see Description (11)]. Patients treated with this product may n=221 n=148 develop hypersensitivity to these non-human mammalian proteins. Gastrointestinal Disorders Hypersensitivity reactions may also be related to antibodies against Abdominal pain 9 (4%) 5 (3%) polyethylene glycol (PEG) [see Warnings and Precautions (5.3)]. Nausea 9 (4%) 8 (5%) 5.2 Neutralizing Antibodies Vomiting 10 (5%) 5 (3%) Neutralizing antibody (inhibitor) formation can occur following General Disorders and administration of Jivi. Carefully monitor patients for the development Administration Site Conditions of Factor VIII inhibitors, using appropriate clinical observations and a 4 (2%) 2 (1%) laboratory tests. If expected plasma Factor VIII activity levels are not Injection site reactions Pyrexia (fever) 20 (9%) 8 (5%) attained or if bleeding is not controlled as expected with administered Immune System Disorders dose, suspect the presence of an inhibitor (neutralizing antibody) Hypersensitivity 8 (4%) 3 (2%) [see Warnings and Precautions (5.4)]. Nervous System Disorders 5.3 Immune Response to PEG 3 (1%) 3 (2%) A clinical immune response associated with IgM anti-PEG antibodies, Dizziness Dysgeusia (distorted sense of taste) 1 (1%) 0 manifested as symptoms of acute hypersensitivity and/or loss of Headache 29 (13%) 21 (14%) drug effect, has been observed primarily in patients < 6 years of age [see Warnings and Precautions (5.1) and Use in Specific Populations Psychiatric Disorders (8.4)]. The symptoms of the clinical immune response were Insomnia 5 (2%) 4 (3%) transient. Anti-PEG IgM titers decreased over time to undetectable Respiratory, Thoracic and levels. No immunoglobulin class switching was observed. Mediastinal Disorders In case of clinical suspicion of loss of drug effect, conduct testing Cough 18 (8%) 10 (7%) for Factor VIII inhibitors [see Warnings and Precautions (5.4) and Skin and Subcutaneous Tissue Adverse Reactions (6.1)] and Factor VIII recovery. Disorders A low post-infusion Factor VIII level in the absence of detectable Erythema b (redness) 3 (1%) 2 (1%) Factor VIII inhibitors indicates that loss of drug effect is likely due Pruritus (itching) 2 (1%) 1 (1%) to anti-PEG antibodies. Discontinue Jivi and switch patients to a Rash c 9 (4%) 3 (2%) previously effective Factor VIII product. B:17 T:15 S:1