Two years of pegvaliase in Germany: Experiences and best practice recommendations
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Two years of pegvaliase in Germany: Experiences and best practice recommendations. / Krämer, Johannes; Baerwald, Christoph; Heimbold, Christian; Kamrath, Clemens; Parhofer, Klaus G; Reichert, Anja; Rutsch, Frank; Stolz, Simone; Weinhold, Natalie; Muntau, Ania C.
In: MOL GENET METAB, Vol. 139, No. 1, 05.2023, p. 107564.Research output: SCORING: Contribution to journal › SCORING: Journal article › Research › peer-review
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TY - JOUR
T1 - Two years of pegvaliase in Germany: Experiences and best practice recommendations
AU - Krämer, Johannes
AU - Baerwald, Christoph
AU - Heimbold, Christian
AU - Kamrath, Clemens
AU - Parhofer, Klaus G
AU - Reichert, Anja
AU - Rutsch, Frank
AU - Stolz, Simone
AU - Weinhold, Natalie
AU - Muntau, Ania C
N1 - Copyright © 2023 BioMarin Deutschland GmbH and BioMarin Pharmaceutical Inc. Published by Elsevier Inc. All rights reserved.
PY - 2023/5
Y1 - 2023/5
N2 - BACKGROUND: In 2019, pegvaliase was approved in Europe for the treatment of phenylketonuria (PKU) in patients aged 16 years and older with blood phenylalanine (Phe) concentrations above 600 μmol/L despite prior management with available treatment options. Since its European approval, German metabolic centres have gained valuable experience, which may be of benefit to other treatment centres managing patients on pegvaliase.METHODS: After a virtual meeting that was attended by nine German physicians, three German dietitians and one American physician, a follow-up discussion was held via an online platform to develop a set of recommendations on the use of pegvaliase in Germany. Eight German physicians contributed to the follow-up discussion and subsequent consensus voting, using a modified Delphi technique. The recommendations were supported by literature and retrospectively collected patient data.RESULTS: Consensus (≥75% agreement) was achieved on 25 recommendations, covering seven topics deemed relevant by the expert panel when considering pegvaliase an option for the treatment of patients with PKU. In addition to the recommendations, a retrospective chart review was conducted in seven of the centres and included 71 patients who initiated treatment with pegvaliase. Twenty-seven patients had been treated for at least 24 months and 23 (85.2%) had achieved blood Phe ≤600 μmol/L with some degree of diet normalisation. Of these patients, 14 had physiological blood Phe on a normalised diet.CONCLUSION: The practical consensus recommendations provide guidance on the different steps along the pegvaliase journey from clinical site requirements to treatment goals and outcomes. The recommendations are intended to support less experienced European metabolic centres with the implementation of pegvaliase, emphasising that a core treatment team consisting of at least a dietitian and metabolic physician is sufficient to initiate pegvaliase and support patients during their treatment journey.
AB - BACKGROUND: In 2019, pegvaliase was approved in Europe for the treatment of phenylketonuria (PKU) in patients aged 16 years and older with blood phenylalanine (Phe) concentrations above 600 μmol/L despite prior management with available treatment options. Since its European approval, German metabolic centres have gained valuable experience, which may be of benefit to other treatment centres managing patients on pegvaliase.METHODS: After a virtual meeting that was attended by nine German physicians, three German dietitians and one American physician, a follow-up discussion was held via an online platform to develop a set of recommendations on the use of pegvaliase in Germany. Eight German physicians contributed to the follow-up discussion and subsequent consensus voting, using a modified Delphi technique. The recommendations were supported by literature and retrospectively collected patient data.RESULTS: Consensus (≥75% agreement) was achieved on 25 recommendations, covering seven topics deemed relevant by the expert panel when considering pegvaliase an option for the treatment of patients with PKU. In addition to the recommendations, a retrospective chart review was conducted in seven of the centres and included 71 patients who initiated treatment with pegvaliase. Twenty-seven patients had been treated for at least 24 months and 23 (85.2%) had achieved blood Phe ≤600 μmol/L with some degree of diet normalisation. Of these patients, 14 had physiological blood Phe on a normalised diet.CONCLUSION: The practical consensus recommendations provide guidance on the different steps along the pegvaliase journey from clinical site requirements to treatment goals and outcomes. The recommendations are intended to support less experienced European metabolic centres with the implementation of pegvaliase, emphasising that a core treatment team consisting of at least a dietitian and metabolic physician is sufficient to initiate pegvaliase and support patients during their treatment journey.
KW - Humans
KW - Retrospective Studies
KW - Phenylalanine Ammonia-Lyase/therapeutic use
KW - Europe
KW - Germany
KW - Phenylketonurias/drug therapy
KW - Phenylalanine
U2 - 10.1016/j.ymgme.2023.107564
DO - 10.1016/j.ymgme.2023.107564
M3 - SCORING: Journal article
C2 - 37086569
VL - 139
SP - 107564
JO - MOL GENET METAB
JF - MOL GENET METAB
SN - 1096-7192
IS - 1
ER -
