Current through Register Vol. 49, No. 9, September, 2024
SECTION I.
DEFINITIONS.
A.
Phenylketonuria (PKU), Congenital
Hypothyroidism (CH) and
Galactosemia
are conditions (diseases) which cause irreversible brain damage
and mental retardation unless they are detected and treated at a very early
stage in the life of a newborn individual. Untreated Galactosemia also results
in liver disease, cataracts, and increased susceptibility to serious infection.
Early diagnosis and treatment are absolutely essential in order to avoid these
health problems.
B.
Sickle Cell Disease (SS) is the most common inherited abnormality
of a red blood cell protein called hemoglobin. It is caused by a genetic
abnormality that must be inherited from both parents. Sickle Cell Disease may
cause serious health problems even in the first few months of life. It occurs
much more commonly in people of African American, Asian and Mediterranean
descent. In addition to the anemia, it lowers resistance to infection, and is
associated with increased morbidity and mortality unless diagnosed and treated
early. Sickle Cell Disease is one of a handful of related hemoglobinopathies
each of which can cause similar health problems with varying
severity.
C.
Sickle Cell
Trait (AS) and other hemoglobinopathy traits differ from their
corresponding diseases. Traits occur when the genetic abnormality is inherited
from only one parent, the other parent contributing a normal gene.
Hemoglobinopathy traits cause only minor health issues that show up
occasionally in life. They are associated with normal life spans. Sickle Cell
Trait (AS) is the most common, occurring in 8 to 10% of African
Americans.
D.
Biotinidase
Deficiency (BIOT) is caused by the lack of an enzyme called biotinidase,
resulting in an inability of the body to use Vitamin B substances absorbed by
the intestines. Without sufficient biotin, several other critical enzyme
systems are unable to function properly. Biotinidase deficiency can lead to
seizures, developmental delay, skin rash, and hearing loss. Newborns with the
disorder appear normal, but develop critical symptoms after the first weeks or
months of life. Symptoms include floppiness, seizures, developmental delay,
hair loss, rashes, hearing loss and vision loss. Metabolic acidosis can result
in coma and death. A daily biotin dietary supplement can prevent all
symptoms.
E.
Congenital
Adrenal Hyperplasia (CAH) is a group of disorders caused by the
deficiency of an adrenal enzyme resulting in decreased production of important
hormones called Cortisol and aldosterone. Cortisol helps the body respond to
stressful events. Aldosterone helps the body maintain its fluids and salts.
Without enough of these hormones, the affected newborn may appear normal, but
can quickly develop symptoms including lethargy, vomiting, muscle weakness and
dehydration. In severe cases death may occur within a few weeks if left
untreated. One kind of CAH may show up first as ambiguous genitalia in the
newborn. Infants with milder forms of the disorder are still at risk for
reproductive and growth difficulties. If detected early and maintained on
appropriate doses of medication, infants diagnosed with CAH can have normal
growth and development.
F.
Cystic Fibrosis (CF) is a disorder in which the body cannot make
an important protein involved in using chloride ions, an ingredient in table
salt. The major clinical consequences are the production of abnormally
thickened mucous secretions in the lungs and digestive systems of affected
newborns. With early detection and lifelong comprehensive treatment plans,
infants diagnosed with CF can be expected to live longer and in a better state
of health than in the past.
G.
Amino Acid Disorders make up a group of inherited conditions in
which protein metabolism is disrupted. Onset of symptoms may occur shortly
after birth or after an apparently normal neonatal period. The symptoms may
occur in episodes with normal periods in between. The clinical onset may
include unusual odors in the urine, irritability, poor feeding, changes in
muscle tone, lightened pigmentation, failure to thrive, jaundice, or liver
enlargement. Other symptoms include intoxication-like symptoms such as
vomiting, lethargy, seizures, and coma. Treatment of amino acid metabolism
disorders includes a low-protein diet strictly controlling intake of specific
amino acids.
H.
Fatty Acid
Oxidation Disorders make up a group of genetic metabolic deficiencies in
which the body is unable to oxidize (break down) fatty acids to make energy. An
enzyme is either missing or not working correctly. The main source of energy
for the body is a sugar called glucose. Normally when the glucose runs out, fat
is broken down into energy. However, that energy is not readily available to
children and adults with a fatty acid oxidation disorder. If undiagnosed and
untreated, these disorders can lead to serious complications affecting the
liver, heart, and eyes; general muscle development; and possibly death.
Symptoms of a metabolic "crisis" include vomiting, diarrhea, lethargy and
difficulty breathing.
IOrganic Acid Disorders make up a group of inherited
metabolic diseases that lead to accumulation of organic acids in biological
fluids (e.g., blood and urine). The accumulation produces disturbances in the
acidity of the body and causes alterations in metabolic chemical reactions.
These disorders can cause intoxication-like symptoms such as vomiting,
metabolic acidosis, ketosis, dehydration, or coma. Some patients may have too
little sugar in the blood, or too much lactic acid or ammonia. Chronic symptoms
include recurrent vomiting, failure to thrive, floppiness and general
developmental delay. Symptoms of these disorders can be diminished by
restricting protein in the diet and, in some cases, supplementation with
vitamins or a nutrient called carnitine.
J.
Severe Combined Immunodeficiency
(SCID) is a group of disorders characterized by severe defects in the
T-lymphocyte and B-lymphocyte systems. Affected babies are susceptible to
multiple types of life-threatening bacterial, viral, and fungal infections.
Early diagnosis of SCID is imperative as SCID is curable with hematopoietic
stem cell transplantation. Infants with SCID die of infections by age two (2)
years unless immunity is reconstituted by treatment. SCID is commonly known as
the "bubble boy" disease.
K. The
Collector is the person or party responsible for collecting and
submitting the blood specimen for testing. The persons or parties who are
collectors under these Rules and Regulations are described in Section
IV.A.
L.
The
Department is the Arkansas Department of Health.
M.
Spinal muscular atrophy (SMA)
is a genetic disease affecting the central nervous system, peripheral nervous
system, and voluntary muscle movement (skeletal muscle). Most of the nerve
cells that control muscles are located in the spinal cord, which accounts for
the word spinal in the name of the disease.
N.
Pompe disease is an inherited
disorder caused by the buildup of a complex sugar called glycogen in the body's
cells. The accumulation of glycogen in certain organs and tissues, especially
muscles, impairs their ability to function normally.
O.
Mucopolysaccharidosis (MPS1)
refers to a group of inherited conditions in which the body is unable to
properly breakdown mucopolysaccharides (long chains of sugar molecules that are
found throughout the body). As a result, these sugars buildup in cells, blood
and connective tissue which can lead to a variety of health problems.
P. Adrenoleukodystrophy (X-ALD) is a disease
linked to the X chromosome. It is a result of fatty acid buildup caused by the
relevant enzymes not functioning properly, which then causes damage to the
myelin sheath of the nerves, resulting in seizures and hyperactivity. Other
symptoms include problems with speaking, listening, and understanding verbal
instructions.
SECTION II.
PURPOSE.
The purpose of these Rules is to assure that all infants born
in Arkansas have the opportunity to be screened for genetic illnesses.
These Rules provide a method to assure that:
1. All newborn infants are tested for
Phenylketonuria (PKU), Congenital Hypothyroidism (CH), Galactosemia, Sickle
Cell Disease (SS), Biotinidase Deficiency (BIOT), Congential Adrenal
Hyperplasia (CAH), Cystic Fibrosis (CF), Amino Acid Disorders, Fatty Acid
Oxidation Disorders, Organic Acid Disorders, and Severe Combined
Immunodeficiency (SCID), Spinal Muscular Atrophy (SMA), Pompe Disease, MPS 1
spectrum of disease, and childhood onset (cerebral) X-ALD.
2. All newborns with an abnormal screen
receive appropriate medical follow-up.
SECTION
IV.
RESPONSIBILITY.
A. Collection and Submission.
1. Medical Facilities/Medical Staff: In all
cases where the birth of an infant occurs in a medical facility licensed by the
Board of Health, it shall be the responsibility of the governing body and
medical staff of the facility to adopt and enforce policies and procedures
which ensures that blood test for Phenylketonuria (PKU), Congenital
Hypothyroidism (CH), Galactosemia, Sickle Cell Disease (SS), Biotinidase
Deficiency (BIOT), Congenital Adrenal Hyperplasia (CAH), Cystic Fibrosis (CF),
Amino Acid Disorders, Fatty Acid Oxidation Disorders, Organic Acid Disorders,
and- Severe Combined Immunodeficiency (SCID), Spinal Muscular Atrophy (SMA),
Pompe Disease, MPS 1 spectrum of disease, and childhood onset (cerebral) X-ALD
are conducted and processed in accordance with these rules. The licensed
facility shall also be responsible for submission of the usable blood specimen
in cases where an infant less than six months of age is admitted (i.e., born
out of hospital, neonatal transfer, etc.), and it is brought to the attention
of the facility or the attending physician that the infant is untested. If an
infant is discharged from a licensed medical facility without collection and
submission of a usable specimen for testing, it shall be the responsibility of
the discharging facility and the attending physician to arrange for the
testing. The discharging facility and attending physician shall notify the
Arkansas Department of Health ("Department") within one week of discharge if
their efforts fail to arrange for testing.
2. Physicians: Physicians assuming care of
infants who are under six months of age and who come to their attention as
being untested or inadequately tested for Phenylketonuria (PKU), Congenital
Hypothyroidism (CH), Galactosemia, Sickle Cell Disease (SS), Biotinidase
Deficiency (BIOT), Congenital Adrenal Hyperplasia (CAH), Cystic Fibrosis(CF),
Amino Acid Disorders, Fatty Acid Oxidation Disorders, Organic Acid Disorders,
and Severe Combined Immunodeficiency (SCID), Spinal Muscular Atrophy (SMA),
Pompe Disease, MPS 1 spectrum of disease, and childhood onset (cerebral) X-ALD,
shall also be responsible for assuring collection and submission of usable
blood specimens for these infants.
3. Licensed Midwives: In cases where the
birth occurs outside a licensed medical facility or in the home, it shall be
the responsibility of an attending licensed midwife to advise the parents of
this law and the procedure for conducting newborn screening, and documenting
that a blood sample is obtained after 24 hours and no later than 72 hours after
birth. If the blood sample is not obtained for any reason, an attending
licensed midwife must document the incident in the patient's chart.
4. The Department: The Department's Local
Health Unit shall collect and submit usable blood specimens on all infants
under six months of age who come to their attention as being tested or
inadequately tested. This responsibility shall not be in lieu of that of the
preceding individuals and facilities.
B. Payment
1. The Collector will be charged a fee of one
hundred and thirty-one dollars ($131.00) for the processing and testing of
newborn screening specimens by the Department.
2. The Board of Health may determine the
amount of this fee based on the Department's cost to process and test the
specimens.
C. Laboratory
Analysis
1. The Department shall be
responsible for provision of forms and instructions for the blood specimen
collection; processing and recording of the specimen received; analysis of
specimen; determination of abnormal results; and reporting of lab results
within a time period which would allow preventive medical
intervention.
D.
Follow-Up
1. The Department shall be
responsible for the interpretation of laboratory results and the reporting of
abnormal results to the attending physician or birth attendant. If the
screening result is suggestive of Classical or Variant PKU, Galactosemia,
Sickle Cell Disease (SS), Biotinidase Deficiency (BIOT), Congenital Adrenal
Hyperplasia (CAH), Cystic Fibrosis (CF), Amino Acid Disorders, Fatty Acid
Oxidation Disorders, Organic Acid Disorders, of Severe Combined
Immunodeficiency (SCID), Spinal Muscular Atrophy (SMA), Pompe Disease, MPS 1
spectrum of disease, or childhood onset (cerebral) X-ALD, the Department shall
consult with specialist physicians who are retained by contract to provide
clinical advice on these conditions. The Department shall notify the Collector
of the specimen and enter the infant's information in a tracking system
maintained to evaluate program operations and infants' medical
outcomes.
2. Attending
Physician/Medical Attendant:
a) Upon receipt
of a notice of an abnormal test result the physician or medical attendant shall
be responsible for the appropriate medical treatment, referral, and/or
retesting within the timeframe specified by the Department for that particular
disorder. It is strongly recommended that consultation be obtained with a
physician who has special competence in the management of these
disorders.
b) The attending
physician or other responsible health care provider who conducts testing in
follow-up to abnormal screens shall report subsequent test results (whether
negative or positive) to the Department. To provide for long term follow up the
Department will collect data on affected infants each year for five years to
determining health care maintenance and health status, especially the presence
of mental retardation or permanent disability.
The Department will establish protocols for follow-up of all
screened disorders in collaboration with medical specialists under contract.
For infants with abnormal test results, the physician will be notified of the
results and informed of the recommended protocols for follow-up of the specific
order.
SECTION V.
SPECIMEN COLLECTION AND
SUBMISSION
A. The blood specimen for
PKU, CH, Galactosemis, Sickle Cell Anemia, Biotinidase (BIOT), Congenital
Adrenal Hyperplasia (CAH), Cystic Fibrosis (CF), Amino Acid Disorders, Fatty
Acid Oxidation Disorders, Organic Acid Disorders, or- Severe Combined
Immunodeficiency (SCID), or Spinal Muscular Atrophy (SMA), Pompe Disease, MPS 1
spectrum of disease, and childhood onset (cerebral) X-ALD testing must be
collected and submitted as described below:
B. Timing of Specimen Collection
1. For all healthy infants born in medical
facilities, the specimen shall be collected before the time of discharge from
the facility. Optimum time for collection is 24 to 72 hours after birth, and
all Collectors should strive to comply with that time frame. If any infant is
discharged or specimen collected prior to 24 hours of age, a repeat test shall
be arranged by the medical facility and the attending physician. This repeat
specimen shall be collected by the infant's seventh day of life. A repeat test
for Sickle Cell Disease shall not be required if specimen was collected prior
to 24 hours of age.
2. Specimens
from ill or premature infants shall be obtained as soon as possible after their
condition has sufficiently stabilized.
3. Specimens from infants not born in medical
facilities shall be collected between 24 and 72 hours after birth.
Infants under six months of age who are known to be untested or
inadequately tested shall have blood specimens collected and submitted by the
responsible authority as soon as possible.
C. Specimen Collection and Submission
1. Specimens shall be dispatched to the
Arkansas Department of Health Public Health Laboratories, Little Rock,
Arkansas, no later than one (1) business day from collection. Specimens are
submitted only on forms provided by the Public Health Laboratory. The Collector
is responsible for supplying complete and accurate identifying information on
the collection form to be used for tracking infants with abnormal screening
results.
D. Forms
1. Submission: forms may be obtained by
writing to the Public Health Laboratories at:
Arkansas Department of Health
201 South Monroe Street
Little Rock, AR 72205
The county health units will not supply these forms.
E. Unsatisfactory
Specimens
1. Inadequate, contaminated, or
otherwise unusable specimens shall be reported to the Collector after
laboratory determination of an unsatisfactory specimen. The Collector shall be
responsible for assuring recollection and resubmission within seven calendar
days of notification.
SECTION VI.
ANALYSIS, INTERPRETATION,
AND REPORTING OF RESULTS
A. Laboratory
Analysis
1. All specimens received by the
laboratory shall be initially examined within five working days of receipt.
Abnormal results shall be reported to the Collector within two working days of
determination.
B.
Interpretations of Results
1. Phenylketonuria
(PKU)
a) The Department shall define the
phenylalanine level which constitutes a positive screening result for
PKU.
b) An infant whose
phenylalanine level is determined by the Department to be negative for PKU
requires no action to be taken. However, attending physicans shall give special
consideration when testing circumstances or infant evaluation/family history
suggests the possibility of need for prescreening in cases where PKU of PKU
variants may actually exist in spite of initial negative screening
results.
2. Congenital
Hypothyroidism (CH)
a) The Department shall
define the thyroxine and thyroid stimulating hormone levels which constitute
positive screening results for CH.
b) Occasionally test results suggestive of CH
may be reported which, upon retesting, will be found within normal limits.
Likewise it is possible that test results which are reported as normal in the
neonatal period could mask the delayed onset of CH. While an infrequent
occurrence, in the face of clinical findings, this possibility must be
considered by the attending physician.
3. Galactosemia
a) The Department shall define the
galactose-1-phosphate uridyl transferase (GALT) levels which constitute
positive screening results for Galactosemia.
b) It is possible that an infant affected
with Galatosemia could have normal initial screening results. This situation is
most likely to occur in infants who have received no or insufficient feedings
with lactose-containing milk or formula prior to testing, or who have received
blood transfusions prior to testing.
c) The medical caretaker shall give special
consideration to retesting any infant whose case findings, testing
circumstances, or family history seems to medically warrant it.
4. Sickle Cell Anemia or Trait
a) The Department shall define the laboratory
value which constitutes a positive screening result for Sickle Cell Disease
(SS), Sickle Cell Trait (AS) or other related hemoglobinopathy.
b) An infant whose hemoglobin is determined
by the Department to be negative for SS or other related serious
hemoglobinopathies requires no special consultation; however, infants with
trait conditions should be followed for mild anemias and urinary tract
infections.
c) The medical
caretaker shall give special consideration to re-testing any infant whose case
findings, testing circumstances, or family history seems to medically warrant
it.
5. Biotinidase
Deficiency (BIOT), Congenital Adrenal Hyperplasia (CAH), Cystic Fibrosis (CF),
Amino Acid Disorders, Fatty Acid Oxygenation Disorders, Organic Acid Disorders,
Severe Combined Immunodeficiency (SCID).
a)
The Department shall define the laboratory value which constitutes a positive
screening result for Biotinidase Deficiency (BIOT), Congenital Adrenal
Hyperplasia (CAH), Cystic Fibrosis (CF), Amino Acid Disorders, Fatty Acid
Oxidation Disorders, Organic Acid Disorders Severe Combined Immunodeficiency
(SCID).
C.
Reporting of Results
1. Phenylketonuria
(PKU), Congenital Hypothyroidisn (CH), Galactosemia Biotinidase Deficiency
(BIOT), Congenital Adrenal Hyperplasia (CAH), Cystic Fibrosis (CF), Amino Acid
Disorders, Fatty Acid Oxidation Disorders, Organic Acid Disorders Severe
Combined Immunodeficiency (SCID).
a)
Immediately upon obtaining the initial positive screening result, the
Department shall notify the attending physician or medical attendant, who shall
be responsible for ensuring that prompt follow-up diagnostic testing is
conducted.
b) Appropriate,
expectant medical management shall not be withheld pending the confirmatory
test results. A non-physician Collector shall immediately refer the infant for
appropriate medical intervention. It is recommended that a pediatric
geneticist, endocrinologist, or pulmonologist consultant be utilized in the
management of these infants.
2. Sickle Cell Disease (SS) and other serious
Hemoglobinopathies
a) Immediately upon
obtaining the initial positive screening result, presumptive of SS or other
serious hemoglobinopathy, the Department shall notify the Collector, who shall
be responsible for insuring that prompt follow-up diagnostic testing is
conducted.
b) Appropriate,
expectant medical management shall not be withheld pending the confirmatory
test results for either SS or other related hemoglobinopathy. Therefore,
non-physician Collector shall immediately refer the infant for appropriate
medical intervention. It is recommended that a pediatric hematologist
consultant be utilized in the management of these infants.
c) Immediately upon obtaining an initial
positive screening, presumptive of trait, the Department shall notify the
Collector in writing. The parent shall be notified in writing by the
Department.
SECTION VII.
ARKANSAS DEPARTMENT OF
HEALTH ROLE IN TREATMENT AND MONITORING
A. Listing of Consultants
1. For Phenylketonuria (PKU), Congenital
Hypothyroidism (CH), Galactosemia, Sickle Cell Disease and other
hemoglobinopathies, Biotinidase Deficiency (BIOT), Congenital Adrenal
Hyperplasia (CAH), Cystic Fibrosis (CF), Amino Acid Disorders, Fatty Acid
Oxidation Disorders, Organic Acid Disorders, Severe Combined Immunodeficiency
(SCID), the Department shall maintain a list of pediatric consultants having
special competence in these disorders, and shall make the names of such
consultants known to the attending physicians of infants with abnormal
screening test results.
B. Registry
1. For Phenylketonuria (PKU), Congenital
Hypothyroidism (CH), Galactosemias, Sickle Cell Disease (SS), and other
hemoglobinopathies, Biotinidase Deficiency (BIOT), Congenital Adrenal
Hyperplasia (CAH), Cystic Fibrosis (CF), Amino Acid Disorders, Fatty Acid
Oxidation Disorders, Organic Acid Disorders, Severe Combined Immunodeficiency
(SCID), the Department shall maintain a registry to record laboratory results
and diagnoses of all tested infants, and to track referral for those infants in
whom abnormal findings were noted during the screening process.
C. Nutritional Therapy
1. Phenylketonuria (PKU)
a) Nutritional therapy with low phenylalanine
formula and/or foods shall be instituted after the diagnosis of PKU.
2. Galactosemia
a) Nutritional therapy with lactose-free
formula and/or foods shall be instituted after the diagnosis of
Galactosemia.
3. Other
genetic conditions
a) Other genetic
conditions discovered by the laboratory testing done pursuant to these
regulations may require nutritional therapy as recommended by specialist
consultants.
SECTION VIII.
SEVERABILITY
If any provision of these Rules, or application thereof to any
person or circumstance is held invalid, such invalidity shall not affect other
provisions or applications of these Rules which give effect without the invalid
provisions or applications, and to this end the provisions here to are declared
to be severable.