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50 Years Ago in The Journal of Pediatrics
Haematopoietic cell transplantation (HCT) in combination with enzyme replacement therapy (ERT) in patients with Hurler syndrome. Bone Marrow Transplant 2006; 38:17-21. 9. Hirth A, Berg A, Greve G. Successful treatment of severe heart failure in an infant with Hurler syndrome. J Inherit Metab Dis 2007;30:820. 10. Tolar J, Grewal SS, Bjoraker KJ, Whitley CB, Shapiro EG, Charnas L, et al. Combination of enzyme replacement and hematopoietic stem cell transplantation as therapy for Hurler syndrome. Bone Marrow Transplant 2007;41:531-5. 11. Grewal SS, Wynn R, Abdenur JE, Burton BK, Gharib M, Haase C, et al. Safety and efficacy of enzyme replacement therapy in combination with hematopoietic stem cell transplantation in Hurler syndrome. Genet Med 2005;7:143-6. 12. Soni S, Hente M, Breslin N, Hersh J, Whitley C, Cheerva A, et al. Pre-stem cell transplantation enzyme replacement therapy in Hurler syndrome does not lead to
significant antibody formation or delayed recovery of the endogenous enzyme post-transplant: a case report. Pediatr Transplant 2007;11:563-7. 13. Vassal G, Michel G, Esperou H, Gentet JC, Valteau-Couanet D, Doz F, et al. Prospective validation of a novel IV busulfan fixed dosing for paediatric patients to improve therapeutic AUC targeting without drug monitoring. Cancer Chemother Pharmacol 2008;61:113-23. 14. Tran HT, Madden T, Petropoulos D, Worth LL, Felix EA, Sprigg-Saenz HA, et al. Individualizing high-dose oral busulfan: prospective dose adjustment in a pediatric population undergoing allogeneic stem cell transplantation for advanced hematologic malignancies. Bone Marrow Transplant 2000;26:463-70. 15. Staba SL, Escolar ML, Poe M, Kim Y, Martin PL, Szabolcs P, et al. Cord-blood transplants from unrelated donors in patients with Hurler’s syndrome. N Engl J Med 2004;350:1960-9.
50 Years Ago in The Journal of Pediatrics CONGENITAL INTERRUPTION
OF THE
AORTIC ARCH
Quie P, Novick R, Adams P, Anderson R, Varco R. J Pediatr 1959;54:87-92
The 1950s marked the height of what many call the “descriptive” phase in the development of pediatric cardiology. Most publications in that time presented the anatomic features of a lesion, usually in a nonsystemic fashion. This report was typical of the era, describing an interrupted aortic arch in 3 infants, 1 of whom was operated on in an unsuccessful attempt at repair. The authors were pioneers who were willing to advance knowledge in a public forum without having had success in helping their patients. Their efforts led to work that improved care, eventually leading to today’s better outcomes. Better diagnostic tools were needed. The cardiac catheterization described in this report used a “urethral catheter” that could be advanced only to the right ventricle, where an angiogram was performed. A second angiogram in the aorta, done a week later, was needed before the diagnosis could be established. Today, the diagnosis would have been made by echocardiography, perhaps even done in utero. Cardiac catheterization may not have been needed before surgery because other imaging modalities (eg, magnetic resonance imaging, computed tomography) would have produced images as good as those produced by angiography. Surgery to repair this type of defect would have focused not only on the aortic arch, but also on the intracardiac lesions that we now know usually accompany an interruption. Echocardiography would have found the ventricular septal defects present in these infants, and repair of these defects would have been an integral part of the operation. Perhaps the most significant improvements in the care of these very sick infants over the past 50 years have occurred in the area of critical care. Today, these infants would have been cared for in an intensive care unit specific to their size, age, and degree of distress. The infant in this report who was brought to the operating room experienced respiratory death because he was not intubated and could not be adequately suctioned. In today’s modern critical care unit, he would have had a controlled airway with constant monitoring of all vital variables, with help available at the bedside to handle any problems that may arise. The authors of this report are to be commended. Their early attempts at diagnosis and therapy helped build the foundation on which modern pediatric cardiology is based. Frank M. Galioto, Jr, MD Child Cardiology Associates Fairfax, Virginia 10.1016/j.jpeds.2008.07.007
Use of Enzyme Replacement Therapy (Laronidase) before Hematopoietic Stem Cell Transplantation for Mucopolysaccharidosis I: Experience in 18 Patients
139
significant antibody formation or delayed recovery of the endogenous enzyme post-transplant: a case report. Pediatr Transplant 2007;11:563-7. 13. Vassal G, Michel G, Esperou H, Gentet JC, Valteau-Couanet D, Doz F, et al. Prospective validation of a novel IV busulfan fixed dosing for paediatric patients to improve therapeutic AUC targeting without drug monitoring. Cancer Chemother Pharmacol 2008;61:113-23. 14. Tran HT, Madden T, Petropoulos D, Worth LL, Felix EA, Sprigg-Saenz HA, et al. Individualizing high-dose oral busulfan: prospective dose adjustment in a pediatric population undergoing allogeneic stem cell transplantation for advanced hematologic malignancies. Bone Marrow Transplant 2000;26:463-70. 15. Staba SL, Escolar ML, Poe M, Kim Y, Martin PL, Szabolcs P, et al. Cord-blood transplants from unrelated donors in patients with Hurler’s syndrome. N Engl J Med 2004;350:1960-9.
50 Years Ago in The Journal of Pediatrics CONGENITAL INTERRUPTION
OF THE
AORTIC ARCH
Quie P, Novick R, Adams P, Anderson R, Varco R. J Pediatr 1959;54:87-92
The 1950s marked the height of what many call the “descriptive” phase in the development of pediatric cardiology. Most publications in that time presented the anatomic features of a lesion, usually in a nonsystemic fashion. This report was typical of the era, describing an interrupted aortic arch in 3 infants, 1 of whom was operated on in an unsuccessful attempt at repair. The authors were pioneers who were willing to advance knowledge in a public forum without having had success in helping their patients. Their efforts led to work that improved care, eventually leading to today’s better outcomes. Better diagnostic tools were needed. The cardiac catheterization described in this report used a “urethral catheter” that could be advanced only to the right ventricle, where an angiogram was performed. A second angiogram in the aorta, done a week later, was needed before the diagnosis could be established. Today, the diagnosis would have been made by echocardiography, perhaps even done in utero. Cardiac catheterization may not have been needed before surgery because other imaging modalities (eg, magnetic resonance imaging, computed tomography) would have produced images as good as those produced by angiography. Surgery to repair this type of defect would have focused not only on the aortic arch, but also on the intracardiac lesions that we now know usually accompany an interruption. Echocardiography would have found the ventricular septal defects present in these infants, and repair of these defects would have been an integral part of the operation. Perhaps the most significant improvements in the care of these very sick infants over the past 50 years have occurred in the area of critical care. Today, these infants would have been cared for in an intensive care unit specific to their size, age, and degree of distress. The infant in this report who was brought to the operating room experienced respiratory death because he was not intubated and could not be adequately suctioned. In today’s modern critical care unit, he would have had a controlled airway with constant monitoring of all vital variables, with help available at the bedside to handle any problems that may arise. The authors of this report are to be commended. Their early attempts at diagnosis and therapy helped build the foundation on which modern pediatric cardiology is based. Frank M. Galioto, Jr, MD Child Cardiology Associates Fairfax, Virginia 10.1016/j.jpeds.2008.07.007
Use of Enzyme Replacement Therapy (Laronidase) before Hematopoietic Stem Cell Transplantation for Mucopolysaccharidosis I: Experience in 18 Patients
139