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Neonatal Hemochromatosis Medication

  • Author: Jatinder Bhatia, MBBS, FAAP; Chief Editor: Carmen Cuffari, MD  more...
 
Updated: Jul 08, 2016
 

Medication Summary

Few drugs are available for treating neonatal hemochromatosis. Infants with neonatal hemochromatosis have been treated with a combination of antioxidants, cryoprotective agents, and chelation.

Three antioxidants are used throughout the course of therapy: N -acetylcysteine, alpha-tocopherol polyethylene glycol succinate (TPGS), and selenium. These are used in combination with prostaglandin E and deferoxamine, which have a cryoprotective effect and which chelate iron, respectively.

A suggested cocktail is the following: N -acetylcysteine 200 mg/kg/d PO divided tid for 17-21 doses, alpha-TPGS 25 IU/kg/d PO divided bid for 6 weeks, deferoxamine 30 mg/kg/d IV infused over 8 hours until the serum ferritin level is less than 500 mcg/L, selenium 3 mcg/kg/d IV continuous infusion for the length of hospitalization, and prostaglandin E1 0.4 mcg/kg/h IV increased to 0.6 mcg/kg/h over 3-4 hours. The infusion is maintained for 10 days.[10]

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Chelating agents

Class Summary

These agents inhibit toxin by reacting with it to form less active or inactive complex. The complex is then excreted from the body.

Deferoxamine (Desferal)

 

Deferoxamine is freely soluble in water. Approximately 8 mg of iron is bound by 100 mg. It is excreted in urine and bile and produces red discoloration of urine. This agent readily chelates iron from ferritin and hemosiderin but not transferrin. It is most effective with continuous infusion. It may be administered IM, SC, or slow IV infusion. Deferoxamine does not effectively chelate other trace metals of nutritional importance.

Vials contain 500 mg of lyophilized sterile drug; add 2 mL sterile water for injection to each vial, bringing concentration to 250 mg/mL. For IV use, may be diluted in 0.9% sterile saline, dextrose 5% in water (D5W), or Ringer solution. IM administration is preferred except in patients with hypotension and cardiovascular collapse, in whom IV should be considered.

The use of deferoxamine in treatment of neonatal hemochromatosis is controversial. Experience is limited; therefore, use with caution.

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Antioxidants

Class Summary

These agents protect sensitive tissues throughout the body from oxidizing substances known as free radicals. Although antioxidants protect most cell membranes, vitamin E is particularly important in preventing damage to the linings of blood vessels and maintaining good circulation.

N-Acetylcysteine (Acetadote)

 

This agent is a derivative of amino acid cysteine and scavenger of oxygen free radicals. It is also a glutathione precursor and is used to replenish depleted intracellular glutathione. Therefore, it theoretically augments antioxidant defenses.

Alpha-tocopherol (Aquasol-E, E-Gems, Ester-E)

 

Vitamin E is particularly important in preventing damage to linings of blood vessels and maintaining good circulation. It acts as an antioxidant in cell membranes to prevent propagated oxidation of unsaturated fatty acids. It is also known to impair hematologic response to iron.

Selenium (Selenicaps, Selenimin)

 

Selenium is an essential trace element, part of the enzyme glutathione peroxidase. It protects cell components from oxidative damage due to peroxides produced in cellular metabolism.

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Prostaglandins

Class Summary

These agents elicit cryoprotective effect.

Alprostadil (Prostin VR Pediatric)

 

Alprostadil (prostaglandin E1) is used primarily to keep patency of ductus arteriosus but also has a mild pulmonary vasodilatory effect. It is reported to inhibit macrophage activation, neutrophil chemotaxis, and release of oxygen radicals and lysosomal enzymes. It affects coagulation by inhibiting platelet aggregation and possibly by inhibiting activation of factor X. It may promote fibrinolysis by stimulating production of tissue plasminogen activator.

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Surfactants

Class Summary

Exogenous surfactant can be helpful in treatment of airspace disease (eg, respiratory distress syndrome [RDS]). After inhaled administration, surface tension is reduced and alveoli are stabilized, decreasing the work of breathing and increasing lung compliance.

Beractant (Survanta)

 

This agent mimics the surface tension–lowering properties of natural lung surfactant. It contains colfosceril palmitate, cetyl alcohol, and tyloxapol. It is used for prophylaxis of RDS in premature infants with birthweight < 1350 g or RDS in premature infants with birthweight >1350 g who have evidence of pulmonary immaturity. It is also used for rescue treatment of infants who develop RDS.

Calfactant (Infasurf)

 

This is a natural bovine calf lung extract containing phospholipids, fatty acids, and surfactant-associated proteins B (260 mcg/mL) and C (390 mcg/mL). Surfactant is an endogenous complex of lipids and proteins that lines alveolar walls and promotes alveolar stability by reducing surface tension. Relative surfactant deficiency is variably present in many lung diseases.

Poractant alfa (Curosurf)

 

This agent lines alveolar walls and promotes alveolar stability against collapse by reducing surface tension at the air-liquid interface of the alveoli.

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Inotropic agents

Class Summary

Positive inotropic agents increase the force of contraction of the myocardium and are used to treat acute and chronic congestive heart failure (CHF). Some may also increase or decrease the heart rate (ie, positive or negative chronotropic agents), provide vasodilatation, or improve myocardial relaxation. These additional properties influence the choice of drug for specific circumstances.

Dopamine

 

Dopamine is used to treat hypotension that is not secondary to hypovolemia. It has a preferential sparing effect on the renal circulation. It is often used with dobutamine.

Dopamine stimulates adrenergic and dopaminergic receptors. Its hemodynamic effect depends on dose. Low doses predominantly stimulate dopaminergic receptors that in turn produce renal and mesenteric vasodilation. High doses produce cardiac stimulation and renal vasodilation.

The mechanism of action of dopamine in neonates is controversial because of variations in endogenous norepinephrine stores, receptor function, and ability to increase stroke volume.

Low doses (< 2 mcg/kg/min) provide dopaminergic stimulation and increases urine output, fractional excretion of sodium, and creatinine clearance.

Intermediate doses (2-10 mcg/kg/min) increase cardiac contractility and blood pressure at low doses and increases heart rate at high doses. Inotropic response varies with gestational age and baseline stroke volume.

High doses (>20 mcg/kg/min) predominantly increase systemic and pulmonary vascular resistance. Use with caution in patients with persistent pulmonary hypertension of the newborn.

Dobutamine

 

Dobutamine provides inotropic support in patients with shock and hypotension. It is not a pressor. This agent is used for demonstrated or suspected decreased cardiac contractility. It is often used in concert with dopamine. Echocardiography is useful in evaluating need (eg, contractility, ventricular dilation, ejection fraction).

Dobutamine produces vasodilation and increases the inotropic state. At high dosages, it may increase heart rate. Onset of action is 1-2 min, peak effect in 10 min. Administer as continuous IV infusion because half-life is several minutes. It is metabolized in the liver.

Dobutamine acts as a synthetic catecholamine with primarily beta1-adrenergic activity. It increases myocardial contractility, cardiac index, oxygen delivery, and oxygen consumption. It decreases systemic and pulmonary vascular resistance in adults.

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Contributor Information and Disclosures
Author

Jatinder Bhatia, MBBS, FAAP Professor of Pediatrics, Medical College of Georgia, Georgia Regents University; Chief, Division of Neonatology, Director, Fellowship Program in Neonatal-Perinatal Medicine, Director, Transport/ECMO/Nutrition, Vice Chair, Clinical Research, Department of Pediatrics, Children's Hospital of Georgia

Jatinder Bhatia, MBBS, FAAP is a member of the following medical societies: American Academy of Pediatrics, American Association for the Advancement of Science, American Pediatric Society, American Society for Nutrition, American Society for Parenteral and Enteral Nutrition, Academy of Nutrition and Dietetics, Society for Pediatric Research, Southern Society for Pediatric Research

Disclosure: Serve(d) as a director, officer, partner, employee, advisor, consultant or trustee for: Gerber.

Coauthor(s)

Roland L Boyd, DO Neonatologist, Section of Neonatology, Neonatal Services, Ltd

Roland L Boyd, DO is a member of the following medical societies: American Academy of Osteopathy, American Academy of Pediatrics, American College of Osteopathic Pediatricians

Disclosure: Nothing to disclose.

Specialty Editor Board

Mary L Windle, PharmD Adjunct Associate Professor, University of Nebraska Medical Center College of Pharmacy; Editor-in-Chief, Medscape Drug Reference

Disclosure: Nothing to disclose.

David A Piccoli, MD Chief of Pediatric Gastroenterology, Hepatology and Nutrition, The Children's Hospital of Philadelphia; Professor, University of Pennsylvania School of Medicine

David A Piccoli, MD is a member of the following medical societies: American Association for the Study of Liver Diseases, American Gastroenterological Association, North American Society for Pediatric Gastroenterology, Hepatology and Nutrition

Disclosure: Nothing to disclose.

Chief Editor

Carmen Cuffari, MD Associate Professor, Department of Pediatrics, Division of Gastroenterology/Nutrition, Johns Hopkins University School of Medicine

Carmen Cuffari, MD is a member of the following medical societies: American College of Gastroenterology, American Gastroenterological Association, North American Society for Pediatric Gastroenterology, Hepatology and Nutrition, Royal College of Physicians and Surgeons of Canada

Disclosure: Received honoraria from Prometheus Laboratories for speaking and teaching; Received honoraria from Abbott Nutritionals for speaking and teaching.

Additional Contributors

Hisham Nazer, MB, BCh, FRCP, , DTM&H Professor of Pediatrics, Consultant in Pediatric Gastroenterology, Hepatology and Clinical Nutrition, University of Jordan Faculty of Medicine, Jordan

Hisham Nazer, MB, BCh, FRCP, , DTM&H is a member of the following medical societies: American Association for Physician Leadership, Royal College of Paediatrics and Child Health, Royal College of Surgeons in Ireland, Royal Society of Tropical Medicine and Hygiene, Royal College of Physicians and Surgeons of the United Kingdom

Disclosure: Nothing to disclose.

References
  1. Adams PC, Searle J. Neonatal hemochromatosis: a case and review of the literature. Am J Gastroenterol. 1988 Apr. 83(4):422-5. [Medline].

  2. Muller-Berghaus J, Knisely AS, Zaum R, et al. Neonatal haemochromatosis: report of a patient with favourable outcome. Eur J Pediatr. 1997 Apr. 156(4):296-8. [Medline].

  3. Colletti RB, Clemmons JJ. Familial neonatal hemochromatosis with survival. J Pediatr Gastroenterol Nutr. 1988 Jan-Feb. 7(1):39-45. [Medline].

  4. McKusick VA. Mendelian Inheritance in Man. 5th ed. Baltimore, Md: Johns Hopkins University Press; 1978.

  5. Whitington PF. Gestational alloimmune liver disease and neonatal hemochromatosis. Semin Liver Dis. 2012 Nov. 32(4):325-32. [Medline].

  6. Liet JM, Urtin-Hostein C, Joubert M, et al. Neonatal hemochromatosis [in French]. Arch Pediatr. 2000 Jan. 7(1):40-4. [Medline].

  7. Whitington PF, Hibbard JU. High-dose immunoglobulin during pregnancy for recurrent neonatal haemochromatosis. Lancet. 2004 Nov 6-12. 364(9446):1690-8. [Medline].

  8. Sigurdsson L, Reyes J, Kocoshis SA, et al. Neonatal hemochromatosis: outcomes of pharmacologic and surgical therapies. J Pediatr Gastroenterol Nutr. 1998 Jan. 26(1):85-9. [Medline].

  9. Babor F, Hadzik B, Stannigel H, Mayatepek E, Hoehn T. Successful management of neonatal hemochromatosis by exchange transfusion and immunoglobulin: a case report. J Perinatol. 2013 Jan. 33(1):83-5. [Medline].

  10. Whitington PF, Kelly S, Ekong UD. Neonatal hemochromatosis: fetal liver disease leading to liver failure in the fetus and newborn. Pediatr Transplant. 2005 Oct. 9(5):640-5. [Medline].

 
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