Pediatric Endocrine Society and Endocrine Society. Treatment of Primary Congenital Hypothyroidism

Transcription

1 Pediatric Endocrine Society and Endocrine Society Treatment of Primary Approved September 2014 Pediatric Endocrine Society 6728 Old McLean Village Drive McLean, VA (P) (F) pedsendo.org Endocrine Society 2055 L Street, NW, Suite 600 Washington, DC (P) (F) endocrine.org

2 Task Force Members Rosalind Brown, MD, Chair M. Carol Greenlee, MD Stephen H. LaFranchi, MD Dana R. Nason, MD Erinn Rhodes, MD, MPH Susan R. Rose, MD James Rosenzweig, MD Ari J. Wassner, MD Task Force Staff Ailene Cantelmi, Associate Director, Assessment, Accreditation & Online Learning (ES) Tonya McFadden, MSA, Manager, CME Accreditation & Practice Improvement (ES) Rebecca Kresowik, Measure Development Consultant Purpose of Measures The Pediatric Endocrine Society and the Endocrine Society formed a Congenital Hypothyroidism Performance Measures Task Force to identify and define the appropriate treatment for patients with primary congenital hypothyroidism. The diagnosis of primary congenital hypothyroidism is based on confirmatory serum testing that shows an abnormally elevated TSH for age. Measure #1: Levothyroxine Treatment Measure #2: Parent Education Measure #3a: Appropriate TSH Monitoring Measure #3b: Appropriate FreeT4 (or Total T4) Monitoring Measure #4: Appropriate FreeT4 (or Total T4) Values Measure #5: Appropriate TSH Values Measure #6: TSH Measurement Following Dosage Change Note: Measures #3a and #3b are a composite measure and must be used together Development and Use of Performance Measures These performance measures were developed using the evidenced-based clinical practice guidelines available for the treatment of primary congenital hypothyroidism. This measurement set is based on the following clinical guidelines: Leger J, Olivieri A, Donaldson M, et al. European Society for Paediatric Endocrinology Consensus Guidelines on screening, diagnosis, and management of congenital hypothyroidism. J Clin Endocrinol Metab; Published online January 21,

3 Rose SR, Brown, RS, et al. AAP, ATA, PES update of newborn screening and therapy for congenital hypothyroidism. Pediatrics 2006; 117: The performance measures found in this document have been developed to, enable the physician to track his or her performance in individual patient care across patient populations. Please note that treatment must be based on individual patient needs and professional judgment. These clinical performance measures and related data specifications, which the Pediatric Endocrine Society and the Endocrine Society developed for an American Board of Pediatric PIM Practice Improvement Module (PIM), are designed for individual physician quality improvement only, are not clinical guidelines and do not establish a standard of medical care. 2

4 Treatment of Primary Measure # 1: Levothyroxine Treatment Numerator: Number of patients who were started on levothyroxine (L-T4) within two weeks of birth or within 24 hours following confirmatory laboratory diagnosis Denominator: All patients ages one month to 36 months with a diagnosis of primary congenital hypothyroidism Measure Statement: The percentage of patients ages one month to 36 months with a diagnosis of primary congenital hypothyroidism who were started on levothyroxine (L-T4) within two weeks of birth or within 24 hours following confirmatory laboratory diagnosis L-T4 treatment should be initiated as soon as possible and no later than 2 weeks after birth or immediately after confirmatory serum test results. (1++) (ESPE 2014) 1 Rationale for the Measure: Optimal cognitive outcome depends on starting treatment early in life. (AAP/ATA/PES) 2 Safe Timely 3

5 Treatment of Primary Measure #2: Parent Education Numerator: Number of patients whose parents/guardians are provided at the first visit after diagnosis with education and written instructions on all of the following: 1) how to administer levothyroxine (L-T4), 2) the importance of regular monitoring of thyroid function tests, and 3) the relationship between good control and cognitive outcome Denominator: All patients ages one month to 36 months with a diagnosis of primary congenital hypothyroidism Measure Statement: The percentage of patients ages one month to 36 months with a diagnosis of primary congenital hypothyroidism whose parents/guardians are provided at the first visit after diagnosis with education and written instructions on all of the following: 1) how to administer levothyroxine (L-T4), 2) the importance of regular monitoring of thyroid function tests, and 3) the relationship between good control and cognitive outcome Parents should be provided with written instructions on L-T4 treatment to avoid uncertainties that might hinder compliance. (1+) (ESPE 2014) 1 Rationale for the Measure: Nonadherence to the treatment is the most common cause of persistent TSH elevation and should be excluded. (AAP/ATA/PES) 2 Safe Patient-Centered 4

6 Treatment of Primary Measure #3a: Appropriate TSH Monitoring (Measures #3a and #3b are a composite measure and must be used together) Numerator: Number of patients who had a TSH measurement performed according to the following schedule: At least every 1 to 3 months for ages birth to 12 months once TSH is normalized At least every 2 to 4 months for ages 12 months to 36 months Denominator: All patients ages one month to 36 months with a diagnosis of primary congenital hypothyroidism Measure Statement: The percentage of patients ages one month to 36 months with a diagnosis of primary congenital hypothyroidism who had a TSH measurement performed according to the following schedule: At least every 1 to 3 months for ages birth to 12 months once TSH is normalized At least every 2 to 4 months for ages 12 months to 36 months The monitoring of L-T4 treatment should be based on periodic measurements of serum or plasma FT4 or TSH concentrations. The first follow-up examination should take place 1-2 weeks after the start of L-T4 treatment. Subsequent evaluation should take place every 2 weeks until a complete normalization of TSH concentration is reached; then every 1 to 3 months thereafter until the age of 12 months. Between the ages of one and three years, children should undergo frequent clinical and laboratory evaluations (every 2 to 4 months). (1+) (ESPE 2014) 1 Rationale for the Measure: Infants need to undergo frequent laboratory and clinical evaluations of thyroid function. (AAP/ATA/PES) 2 Safe Timely Effective 5

7 Treatment of Primary Measure #3b: Appropriate FreeT4 (or Total T4) Monitoring (Measures #3a and #3b are a composite measure and must be used together) Numerator: Number of patients who had a freet4 (or total T4) measurement performed according to the following schedule: At least every 1 to 3 months for ages birth to 12 months once freet4 (or total T4) is normalized At least every 2 to 4 months for ages 12 months to 36 months Denominator: All patients ages one month to 36 months with a diagnosis of primary congenital hypothyroidism Measure Statement: The percentage of patients ages one month to 36 months with a diagnosis of primary congenital hypothyroidism who had a freet4 (or total T4) measurement performed according to the following schedule: At least every 1 to 3 months for ages birth to 12 months once freet4 (or total T4) is normalized At least every 2 to 4 months for ages 12 months to 36 months The monitoring of L-T4 treatment should be based on periodic measurements of serum or plasma FT4 or TSH concentrations. The first follow-up examination should take place 1-2 weeks after the start of L-T4 treatment. Subsequent evaluation should take place every 2 weeks until a complete normalization of TSH concentration is reached; then every 1 to 3 months thereafter until the age of 12 months. Between the ages of one and three years, children should undergo frequent clinical and laboratory evaluations (every 2 to 4 months). (1+)(ESPE 2014) 1 Rationale for the Measure: Infants need to undergo frequent laboratory and clinical evaluations of thyroid function. (AAP/ATA/PES) 2 Safe Timely Effective 6

8 Treatment of Primary Measure #4: Appropriate FreeT4 (or Total T4) Values Numerator: Number of patients whose most recent measured free T4 (or total T4) concentration was within the upper half of the assay reference range OR if free T4 (or total T4) was not within the upper half of the assay reference range an adjustment to levothyroxine (L-T4) was made Within the upper half of the assay reference range defined: Free T4 or total T4 concentration at or between the midpoint and the upper limit of the assay-specific normal range. The midpoint of the normal range is the mean of the upper and lower limits of the normal range. Denominator: All patients ages one month to the 19 th birthday with a diagnosis of primary congenital hypothyroidism Measure Statement: The percentage of patients ages one month to the 19 th birthday with a diagnosis of primary congenital hypothyroidism whose most recent measured free T4 (or total T4) concentration was within the upper half of the assay reference range OR if free T4 (or total T4) was not within the upper half of the assay reference range an adjustment to levothyroxine (L-T4) was made TT4 or FT4 concentration should be maintained in the upper half of the age-specific reference range. (1++) (ESPE 2014) 1 Rationale for the Measure: The aim of therapy is to ensure normal growth and development by maintaining the serum T 4 or FT 4 concentration in the upper half of the reference range. (AAP/ATA/PES) 2 Dynamic changes in the T4 (and free T4) concentrations occur particularly in the first 1-2 weeks of life. 3, 4 The task force determined that since free T 4 values vary significantly between assays, the measure would focus on the upper half of the assay-specific rather than age-specific reference range. Effective 7

9 Treatment of Primary Measure #5: Appropriate TSH Values Numerator: Number of patients whose most recent measured TSH value was in the range mu/l OR if TSH was not in the range mu/l an adjustment to levothyroxine (L-T4) was made Denominator: All patients ages one month to the 19 th birthday with a diagnosis of primary congenital hypothyroidism Measure Statement: The percentage of patients ages one month to the 19 th birthday with a diagnosis of primary congenital hypothyroidism whose most recent measured TSH value was in the range mu/l OR if TSH was not in the range mu/l an adjustment to levothyroxine (L-T4) was made TSH concentration should be maintained in the age-specific reference range (but to avoid undetectable TSH < 0.05 mu/l). (1++) (ESPE 2014) 1 Rationale for the Measure: The aim of therapy is to ensure normal growth and development by maintaining the serum TSH in the desired range. (AAP/ATA/PES) 2 Dynamic changes in the TSH concentration occur particularly in the first few days of life with a gradual deceleration thereafter. 3, 4 By 1 month of age a TSH concentration <5 mu/l is found in 97.5% of infants. After review of the evidence, the task force determined that the TSH concentration range for the measure would be mu/l. Effective 8

10 Treatment of Primary Measure #6: TSH Measurement Following Dosage Change Numerator: Number of patients who had a TSH measurement performed within 4 8 weeks after the most recent levothyroxine (L-T4) dosage change Denominator: All patients ages one month to the 19 th birthday with a diagnosis of primary congenital hypothyroidism Measure Statement: The percentage of patients ages one month to the 19 th birthday with a diagnosis of primary congenital hypothyroidism who had a TSH measurement performed within 4 8 weeks after the most recent levothyroxine (L-T4) dosage change TSH measurements should be performed 4 to 6 weeks after any change in L-T4 dose or L-T4 formulation (eg, switch from brand to generic L-T4). (1+) (ESPE 2014) 1 Rationale for the Measure: The aim of therapy is to ensure normal growth and development by maintaining the serum TSH in the desired range. (AAP/ATA/PES) 2 Safe Timely Effective 9

11 ESPE Guidelines Rating System Rating Recommendation Definition 1 Strong Recommendation 2 Weak Recommendation Applies to most patients in most circumstances, benefits clearly outweigh the risk Consensus opinion of working group or should be considered; the best action may depend on circumstances or patient values, benefits and risks closely balanced or uncertain Evidence +++ High Prospective cohort studies or randomized controlled trials at low risk of bias ++0 Moderate Observational studies or trials with methodological flaws, inconsistent or indirect evidence +00 Low Case series or non-systematic clinical observations 10

12 References 1 Leger J, Olivieri A, Donaldson M, et al. European Society for Paediatric Endocrinology Consensus Guidelines on screening, diagnosis, and management of congenital hypothyroidism. J Clin Endocrinol Metab; Published online January 21, Rose SR, Brown, RS, et al. AAP, ATA, PES update of newborn screening and therapy for congenital hypothyroidism. Pediatrics 2006; 117: Chaler EA, Fiorenzano R, et al. Age-specific thyroid hormone and thyrotropin reference intervals for a pediatric and adolescent population. Clin Chem Lab Med 2012; 50(5): Elmlinger MW, Kuhnel W, et al. Reference intervals from birth to adulthood for serum thyroxin (T 4 ) triiodothyronine (T 3 ), free T 3, free T 4, thyroxine binding globulin (TBG) and thyrotropin (TSH). Clin Chem Lab Med 2001; 39(10):