Journal of Pediatric Rehabilitation Medicine: An Interdisciplinary Approach 7 (2014) 107–109 DOI 10.3233/PRM-140279 IOS Press
107
Guest editorial
Bone health in children with special health care needs
Since its’ launch in 2008, the Journal of Pediatric Rehabilitation Medicine (JPRM) has been generous in publishing articles focused on bone health in children with special needs [1–4]. However, to my knowledge, outside of conference proceedings, there has yet to be an entire issue dedicated to this subject in this, or in any pediatric focused medical journal. Perhaps it is because pediatric bone densitometry is a relatively new field, initiated in the mid-1990s when it became clear that osteoporosis had its origins in the growing child. Alternatively, the scarcity of publications maybe related to the fact that there are few published guidelines for how to care for bone health of children, much less children with special needs. In 2007, the first textbook was published on Guidelines for Pediatric Bone Densitometry [5]. One year later, the International Society for Clinical Densitometry set standards for assessment and developed training courses in pediatric bone densitometry [6]. Neither of these guides had a focus on the child with special health care needs. Clearly these children have an increased risk for fracture from minimal trauma, and we, as their health care providers need some practical guidelines for how to proceed. Therefore, I am pleased to welcome you to this special edition of JPRM, focused on bone health for children with special health care needs. The aim of this issue is to review the occurrence of bone health issues in children with neuromuscular diseases and limited mobility, describe the risk factors associated with them, and when available, provide a summary of the evidenced based treatments. I commend these authors for their work. In many cases there was a paucity of literature to review. That which was available, was often performed in small case series, not the gold standard RCT trials necessary to support clinical decisions. However, these authors thoroughly reviewed their respective fields and did their best to skillfully judge the data that was available.
The first manuscript addresses the assessment of bone health in these patients. In the last decade it has become clear that assessment of bone health in children is different from adults, and there are a number of scientists focused on how best to assess children, particularly those who are small for their age [7, 8]. But children with special health care needs have distinct challenges with respect to bone health assessment: attention deficits, cognitive delays, startle reflexes, significant contractures and severe scoliosis, dislocated hips, orthopedic hardware such as spinal fusion and/or osteotomies, and other artifacts such as g-tubes, tracheostomies and/or baclofen pumps. Ms. Kecskemethy [9] provides a detailed review of the relevant literature and background for the specific techniques that are especially useful for measuring bone density in children with disabilities. The review provides essentials for the technician and clinician alike to consider when assessing and interpreting dual energy x-ray absorptiometry scans. Drs. Houlihan, Apkon and Ness [10,11] take on the task of reviewing the relevant literature regarding bone health in the large numbers of children with Cerebral Palsy (CP) and Neuromuscular Disorders. Though children with CP are probably the best understood of all the pediatric patients with special needs, there remains a paucity of literature regarding how to address very low bone mineral density in these patients, and more specifically, how to keep them from fracturing. Drs. Apkon and Ness elegantly review the latest bone relevant publications in Duchenne muscular dystrophy (DMD) and spinal muscular atrophy (SMA). These patients are at higher risk for low bone mass and fracture compared to their able-bodied peers primarily due to poor muscle tone, limited weight bearing, and use of corticosteroids. Fractures cause pain, loss of mobility, skeletal deformity, and loss of time from school and
c 2014 – IOS Press and the authors. All rights reserved 1874-5393/14/$27.50
108
Guest Editorial
work; therefore interventions for how to avoid fracture in these high-risk groups are being tested and reviewed. Vitamin D is often sub-optimal in patients in rehabilitation units, in particular those with CP and DMD. For years, dietary calcium intake was the primary focus to enhance bone mineralization; it has now become clear that vitamin D is critical for both calcium absorption and optimal bone mineralization from youth through adulthood. Dr. Holick [12], an adult endocrinologist who has been working in the field of vitamin D metabolism for 3 decades, expands our understanding of this vitamin from its’ skeletal to non-skeletal effects. He elucidates why our patients, particularly those on anti-seizure medications are at such risk for deficiency. Skeletal disease causes significant morbidity in patients with mucopolysaccharidosis (MPS) or those with RETT syndrome. The objective, of the first original manuscript in this volume, was to determine if biomarkers of bone turnover were abnormal in children with specific MPS disorders and if they were associated with measures of physical function or pain [13]. The observations of Drs. Polgreen and Stevenson are an exciting addition to this edition and the investigative team is to be commended. Gathering longitudinal data on 39 patients with MPS is a daunting task as it is a truly orphan disease with approximately 1:100,000 new diagnoses each year. In the second original article, Dr. Pico and colleagues performed a pilot singlearm intervention study in a small cohort of patients with RETT syndrome, another rare, neurodevelopmental disorder resulting from spontaneous mutations on the X chromosome [14]. Their study incorporated the use of the vibration platform, which has been shown effective in other pediatric patients with low bone mass previously, in particular, those with cerebral palsy. The therapy, only 6 months in length, focused on just 11 subjects, but shows promise for this at risk patient population, not only for bone health outcomes, but nonskeletal effects such as possible desensitization. Many children with mobility and neuromuscular diseases are at increased risk for fracture either at present or in the future given multiple comorbidities including low body weight, immobilization, seizure medications, fracture and casting history. For some young children with osteogenesis imperfecta (OI), fractures are a painful unpredictable part of everyday life. Conservative dietary and exercise based therapies are ineffective – the only treatment remaining for them is aggressive bone anti-resorptive medications such
as pamidronate and zoledronate. Though many pediatricians are regularly prescribing pamidronte therapy to OI patients without incident, Dr. Olsen provides a cautionary tale of a very young child with OI type I who suffered severe respiratory distress during a second infusion of pamidronate [15]. Though bisphosphonates have been well studied in adults, no large-scale RCT trials have been performed in pediatrics outside of those with OI. However, the use of bisphosphonate medications in children with open growth plates is on the rise, and is cause for concern for many pediatricians. The cost benefit analysis of this issue is addressed in the very thorough and thought provoking review by Dr. Szalay [16]. It has become clear that osteoporosis begins in the pediatric years and that assessment of bone health in children requires different techniques than in adults. Moreover, many children with special health care needs have multiple risk factors for low bone mass and fracture risk. If we are more aware of the causes of low bone mass in these children with special health care needs, we may be able to lessen their risk of osteoporosis in the present day as well as later in life. This understanding could hopefully minimize an individuals need for more invasive therapies, such as bisphosphonates as adults. Respectfully Submitted, Ellen B. Fung, PhD, RD Associate Research Scientist Children’s Hospital Oakland Research Institute E-mail: [email protected] References [1]
Editorial: Pediatric Limb deficiencies. J Pediatr Rehab Med 2009; 2: 3: 151-245. [2] Fung EB, Johnson J, Madden J, Kim T, Harmatz P. Bone density assessment in patients with mucopolysaccharidosis: A preliminary report from patients with MPS II and VI. J Pediatr Rehab Med 2010; 3: 1. [3] Editorial: Promoting Bone Health in MPS VI (MaroteauxLamy syndrome): Framing New Therapies, Part 2. J Pediatr Rehab Med 2010; 3: 2: 83-156. [4] Cohran V, Cassedy A, Hawkins A, Bean J, Heubi J. Oral risedronate sodium improves bone mineral density in nonambulatory patients: A randomized, double-blind, placebo controlled trial. J Pediatr Rehab Med 2013; 6: 2: 85-93. [5] Sawyer AJ, Bachrach LK, Fung EB. (Editors) Bone Densitometry in Growing Patients: Guidelines for Clinical Practice. Humana Press, Totowa, NJ, 2007. [6] Gordon CM, Baim S, Bianchi ML, Bishop NJ, Hans DB, Kalkwarf H, et al. Special report on the 2007 Pediatric Position Development Conference of the International Society for Clinical Densitometry. South Med J 2008 Jul; 101(7): 740-3.
[7] [8]
[9] [10] [11] [12]
Guest Editorial
109
[13]
Polgreen, L, Stevenson, D. Biomarkers of bone remodeling in children with Mucopolysaccaridosis I, II and VI. JPRM, 2014; 7: 2: 159-165. Sarah Y. Afzal, Anna R. Wender, Mary D. Jones, Ellen B. Fung, Elaine L. Pico. The effect of low magnitude mechanical stimulation (LMMS) on bone density in patients with Rett syndrome: A pilot and feasibility study, JPRM 2014; 7(2): 167-178. Olson J. Severe respiratory distress during infusion of pamidronate in a 3 yo male with Osteogenesis Imperfecta. JPRM 2014; 7: 2: 155-158. Szalay E, Bisphosphonate Use in Children with Other Bone Conditions, JPRM 2014; 7(2): 125-132.
Heaney RP. BMC, not BMD is the correct bone measure for growth studies. AJCN 2003; 78: 350-352. Zemel BS, et al. Height adjustment in assessing DXA measurements of Bone mass and density in Children. JCEM 2010; 95: 1265-1273. Kecskemethy H. Assessment of Bone Health in Children with Disabilities. JPRM 2014; 7: 2: 111-124. Houlihan C. Bone Health in Cerebral Palsy: who’s at risk and what to do about it? JPRM 2014; 7: 2: 143-153. Apkon SD and Ness K. Bone health in children with neuromuscular disorders. JPRM 2014; 7: 2: 133-142. Holick M. Vitamin D and Bone Health in Children with Disabilities JPRM 2014; 7: 2: 179-192.
[14]
[15]
[16]
Copyright of Journal of Pediatric Rehabilitation Medicine is the property of IOS Press and its content may not be copied or emailed to multiple sites or posted to a listserv without the copyright holder's express written permission. However, users may print, download, or email articles for individual use.
107
Guest editorial
Bone health in children with special health care needs
Since its’ launch in 2008, the Journal of Pediatric Rehabilitation Medicine (JPRM) has been generous in publishing articles focused on bone health in children with special needs [1–4]. However, to my knowledge, outside of conference proceedings, there has yet to be an entire issue dedicated to this subject in this, or in any pediatric focused medical journal. Perhaps it is because pediatric bone densitometry is a relatively new field, initiated in the mid-1990s when it became clear that osteoporosis had its origins in the growing child. Alternatively, the scarcity of publications maybe related to the fact that there are few published guidelines for how to care for bone health of children, much less children with special needs. In 2007, the first textbook was published on Guidelines for Pediatric Bone Densitometry [5]. One year later, the International Society for Clinical Densitometry set standards for assessment and developed training courses in pediatric bone densitometry [6]. Neither of these guides had a focus on the child with special health care needs. Clearly these children have an increased risk for fracture from minimal trauma, and we, as their health care providers need some practical guidelines for how to proceed. Therefore, I am pleased to welcome you to this special edition of JPRM, focused on bone health for children with special health care needs. The aim of this issue is to review the occurrence of bone health issues in children with neuromuscular diseases and limited mobility, describe the risk factors associated with them, and when available, provide a summary of the evidenced based treatments. I commend these authors for their work. In many cases there was a paucity of literature to review. That which was available, was often performed in small case series, not the gold standard RCT trials necessary to support clinical decisions. However, these authors thoroughly reviewed their respective fields and did their best to skillfully judge the data that was available.
The first manuscript addresses the assessment of bone health in these patients. In the last decade it has become clear that assessment of bone health in children is different from adults, and there are a number of scientists focused on how best to assess children, particularly those who are small for their age [7, 8]. But children with special health care needs have distinct challenges with respect to bone health assessment: attention deficits, cognitive delays, startle reflexes, significant contractures and severe scoliosis, dislocated hips, orthopedic hardware such as spinal fusion and/or osteotomies, and other artifacts such as g-tubes, tracheostomies and/or baclofen pumps. Ms. Kecskemethy [9] provides a detailed review of the relevant literature and background for the specific techniques that are especially useful for measuring bone density in children with disabilities. The review provides essentials for the technician and clinician alike to consider when assessing and interpreting dual energy x-ray absorptiometry scans. Drs. Houlihan, Apkon and Ness [10,11] take on the task of reviewing the relevant literature regarding bone health in the large numbers of children with Cerebral Palsy (CP) and Neuromuscular Disorders. Though children with CP are probably the best understood of all the pediatric patients with special needs, there remains a paucity of literature regarding how to address very low bone mineral density in these patients, and more specifically, how to keep them from fracturing. Drs. Apkon and Ness elegantly review the latest bone relevant publications in Duchenne muscular dystrophy (DMD) and spinal muscular atrophy (SMA). These patients are at higher risk for low bone mass and fracture compared to their able-bodied peers primarily due to poor muscle tone, limited weight bearing, and use of corticosteroids. Fractures cause pain, loss of mobility, skeletal deformity, and loss of time from school and
c 2014 – IOS Press and the authors. All rights reserved 1874-5393/14/$27.50
108
Guest Editorial
work; therefore interventions for how to avoid fracture in these high-risk groups are being tested and reviewed. Vitamin D is often sub-optimal in patients in rehabilitation units, in particular those with CP and DMD. For years, dietary calcium intake was the primary focus to enhance bone mineralization; it has now become clear that vitamin D is critical for both calcium absorption and optimal bone mineralization from youth through adulthood. Dr. Holick [12], an adult endocrinologist who has been working in the field of vitamin D metabolism for 3 decades, expands our understanding of this vitamin from its’ skeletal to non-skeletal effects. He elucidates why our patients, particularly those on anti-seizure medications are at such risk for deficiency. Skeletal disease causes significant morbidity in patients with mucopolysaccharidosis (MPS) or those with RETT syndrome. The objective, of the first original manuscript in this volume, was to determine if biomarkers of bone turnover were abnormal in children with specific MPS disorders and if they were associated with measures of physical function or pain [13]. The observations of Drs. Polgreen and Stevenson are an exciting addition to this edition and the investigative team is to be commended. Gathering longitudinal data on 39 patients with MPS is a daunting task as it is a truly orphan disease with approximately 1:100,000 new diagnoses each year. In the second original article, Dr. Pico and colleagues performed a pilot singlearm intervention study in a small cohort of patients with RETT syndrome, another rare, neurodevelopmental disorder resulting from spontaneous mutations on the X chromosome [14]. Their study incorporated the use of the vibration platform, which has been shown effective in other pediatric patients with low bone mass previously, in particular, those with cerebral palsy. The therapy, only 6 months in length, focused on just 11 subjects, but shows promise for this at risk patient population, not only for bone health outcomes, but nonskeletal effects such as possible desensitization. Many children with mobility and neuromuscular diseases are at increased risk for fracture either at present or in the future given multiple comorbidities including low body weight, immobilization, seizure medications, fracture and casting history. For some young children with osteogenesis imperfecta (OI), fractures are a painful unpredictable part of everyday life. Conservative dietary and exercise based therapies are ineffective – the only treatment remaining for them is aggressive bone anti-resorptive medications such
as pamidronate and zoledronate. Though many pediatricians are regularly prescribing pamidronte therapy to OI patients without incident, Dr. Olsen provides a cautionary tale of a very young child with OI type I who suffered severe respiratory distress during a second infusion of pamidronate [15]. Though bisphosphonates have been well studied in adults, no large-scale RCT trials have been performed in pediatrics outside of those with OI. However, the use of bisphosphonate medications in children with open growth plates is on the rise, and is cause for concern for many pediatricians. The cost benefit analysis of this issue is addressed in the very thorough and thought provoking review by Dr. Szalay [16]. It has become clear that osteoporosis begins in the pediatric years and that assessment of bone health in children requires different techniques than in adults. Moreover, many children with special health care needs have multiple risk factors for low bone mass and fracture risk. If we are more aware of the causes of low bone mass in these children with special health care needs, we may be able to lessen their risk of osteoporosis in the present day as well as later in life. This understanding could hopefully minimize an individuals need for more invasive therapies, such as bisphosphonates as adults. Respectfully Submitted, Ellen B. Fung, PhD, RD Associate Research Scientist Children’s Hospital Oakland Research Institute E-mail: [email protected] References [1]
Editorial: Pediatric Limb deficiencies. J Pediatr Rehab Med 2009; 2: 3: 151-245. [2] Fung EB, Johnson J, Madden J, Kim T, Harmatz P. Bone density assessment in patients with mucopolysaccharidosis: A preliminary report from patients with MPS II and VI. J Pediatr Rehab Med 2010; 3: 1. [3] Editorial: Promoting Bone Health in MPS VI (MaroteauxLamy syndrome): Framing New Therapies, Part 2. J Pediatr Rehab Med 2010; 3: 2: 83-156. [4] Cohran V, Cassedy A, Hawkins A, Bean J, Heubi J. Oral risedronate sodium improves bone mineral density in nonambulatory patients: A randomized, double-blind, placebo controlled trial. J Pediatr Rehab Med 2013; 6: 2: 85-93. [5] Sawyer AJ, Bachrach LK, Fung EB. (Editors) Bone Densitometry in Growing Patients: Guidelines for Clinical Practice. Humana Press, Totowa, NJ, 2007. [6] Gordon CM, Baim S, Bianchi ML, Bishop NJ, Hans DB, Kalkwarf H, et al. Special report on the 2007 Pediatric Position Development Conference of the International Society for Clinical Densitometry. South Med J 2008 Jul; 101(7): 740-3.
[7] [8]
[9] [10] [11] [12]
Guest Editorial
109
[13]
Polgreen, L, Stevenson, D. Biomarkers of bone remodeling in children with Mucopolysaccaridosis I, II and VI. JPRM, 2014; 7: 2: 159-165. Sarah Y. Afzal, Anna R. Wender, Mary D. Jones, Ellen B. Fung, Elaine L. Pico. The effect of low magnitude mechanical stimulation (LMMS) on bone density in patients with Rett syndrome: A pilot and feasibility study, JPRM 2014; 7(2): 167-178. Olson J. Severe respiratory distress during infusion of pamidronate in a 3 yo male with Osteogenesis Imperfecta. JPRM 2014; 7: 2: 155-158. Szalay E, Bisphosphonate Use in Children with Other Bone Conditions, JPRM 2014; 7(2): 125-132.
Heaney RP. BMC, not BMD is the correct bone measure for growth studies. AJCN 2003; 78: 350-352. Zemel BS, et al. Height adjustment in assessing DXA measurements of Bone mass and density in Children. JCEM 2010; 95: 1265-1273. Kecskemethy H. Assessment of Bone Health in Children with Disabilities. JPRM 2014; 7: 2: 111-124. Houlihan C. Bone Health in Cerebral Palsy: who’s at risk and what to do about it? JPRM 2014; 7: 2: 143-153. Apkon SD and Ness K. Bone health in children with neuromuscular disorders. JPRM 2014; 7: 2: 133-142. Holick M. Vitamin D and Bone Health in Children with Disabilities JPRM 2014; 7: 2: 179-192.
[14]
[15]
[16]
Copyright of Journal of Pediatric Rehabilitation Medicine is the property of IOS Press and its content may not be copied or emailed to multiple sites or posted to a listserv without the copyright holder's express written permission. However, users may print, download, or email articles for individual use.
