Childhood analysis

Researchers call for continuous multidisciplinary care into adulthood for Prader-Willi syndrome

Multidisciplinary care that includes growth hormone treatment should not stop once patients with Prader-Willi syndrome reach adulthood, according to the authors of an outcome study.

The combination of growth hormone therapy (GHT) and multidisciplinary care (GHMDC) has been shown in a new study to lead to significantly better health outcomes in adults with Prader-Willi syndrome (PWS) who received this combination of care throughout childhood. Accordingly, such a treatment regimen should be continued after people with PWS reach adulthood, according to new study results Posted in Journal of Clinical Medicine.

There is a high death rate in children and adults with PWS, their hypothalamic dysfunction is also often associated with overeating, pituitary hormone deficiencies, abnormal temperature regulation, and inadequate pain recording. Other somatic and psychosocial factors associated with PWS include musculoskeletal problems, low basal metabolic rate, behavioral problems, biochemical abnormalities, and cardiovascular risk factors (eg, obesity, hypertension, diabetes mellitus. type 2 [T2D]). GHT has been shown to improve many of them, the survey authors noted.

“GHMDC has dramatically improved the health of children with PWS,” the authors wrote. “However, little is known about the effects of childhood GHMDC on health outcomes in adulthood.”

The 109 patients (n = 53, males; 56, females; median age, 28 [range, 18-72] years; median body mass index [BMI], 29 kg / m2) included in the final study analysis received outpatient care at the Dutch Reference Center for Prader-Willi Syndrome in Rotterdam, the Netherlands, between January 2015 and January 2021, and they were divided into 3 groups for this analysis:

  • Received GHMDC from childhood to adulthood (GHMDC + group; n = 39)
  • Never received GHMDC (GHMDC- group; n = 63)
  • Received GHMDC during childhood, but was discontinued before adulthood (GHMDC+ group; n = 7)

Overall, patient screening showed a difference of 74 percentage points in those who reported at least 1 undetected condition between the GHMDC + (10%) and GHMDC– (84%) groups. In addition, no health problem in the GHMDC + group developed between the last pediatric visit and the first adult visit and did not require medical intervention, and no one in the GHMDC + group reported to the doctor. at least 2 or 3 undetected health problems against 41% and 14% in the GHMDC– group.

Significant differences were also observed in the median BMI and prevalence of T2DM in the GHMDC + group compared to the GHMDC– group, even after controlling for age:

  • BMI: 20 (19-24) vs 38 (31-51) kg / m2
  • Incidence of T2DM: 0% vs 27% (including 75% treated)

Meanwhile, the overall rates of hypertension, high cholesterol, and vitamin D deficiency were at least 21 percentage points lower in the GHMDC + group compared to the GHMDC– group.

The authors pointed out that because obesity and T2DM are important risk factors for cardiovascular disease, their discovery of their effects on outcome in people in the GHMDC– group is particularly important. For this reason, the patient’s vulnerability to both must be reduced.

It is also possible that GHMDC will prevent both in patients with PWS and may lead to the early detection of otherwise undiagnosed problems.

“Our data clearly shows that the combination of the two has beneficial effects. However, since all patients who received GHT were treated in a multidisciplinary setting, it is not known which effects are the result of GHT and which are the result of multidisciplinary care, ”the authors concluded. “Therefore, we recommend continuing the GHMDC after patients with PWS reach adulthood. “

Since not all adults living with PWS have access to hormone therapy, the authors also support approval of treatment for this entire group.


Pellikaan K, Rosenberg AG, Davidse K, et al. Effects of multidisciplinary childhood care and growth hormone treatment on health problems in adults with Prader-Willi syndrome. J Clin Med. 2021; 10 (15): 3250. doi: 10.3390 / jcm10153250

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