Pharmacokinetics, Pharmacodynamics, and Pharmacogenomics of Immunosuppressants in Allogeneic Hematopoietic Cell Transplantation: Part II

Bemer, Meagan J.; Long‐Boyle, Janel

doi:10.1007/s40262-015-0340-9

Cited by 33 publications

(31 citation statements)

References 217 publications

(403 reference statements)

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“…This long half-life allows for convenient once-daily dosing, but a loading dose is recommended to achieve target drug concentrations rapidly. 11,12 These pharmacodynamics make mandatory sirolimus monitoring, even more in neonates, in which the drugmetabolizing enzyme systems are in constant development and are still poorly understood. 13 Regarding acute side effects, most of them are dose dependent and improve with dose-reduction or disappear after a few weeks from withdrawal.…”

Section: Sirolimus In Neonates 507mentioning

confidence: 99%

Oral Sirolimus: An Option in the Management of Neonates with Life-Threatening Upper Airway Lymphatic Malformations

Triana

Miguel

Díaz

et al. 2019

Lymphatic Research and Biology

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Background: Mechanistic target of rapamycin (mTOR) inhibitors are being used off-label showing promising results in patients with vascular anomalies. Children with lymphatic malformations (LMs) involving the airway benefit from sirolimus therapy soon after birth, reducing the need of tracheostomy. Available information about efficacy and side effects in neonates remains poor. We present seven newborns with severe head and neck LM showing response to sirolimus with no significant toxicity. Methods and Results: We performed a retrospective review of neonates with head and neck LM who received sirolimus between January 2014 and May 2018 with upper airway involvement needing ventilatory support. We analyzed type of LM, involved anatomical area, symptoms and response to sirolimus, including dosage, blood levels, response, side effects, and complications. Seven neonates received primary treatment with sirolimus in the context of cervical LM. Sirolimus was started at the recommended dose of 0.8 mg/m 2 /12 h and adjusted to maintain blood levels between 4 and 12 ng/mL. Median follow-up was 32 months (4-43) with a median treatment duration of 12 months (3-43). One patient had complete resolution of the malformation, one had complete resolution of symptoms, and five had partial resolution of the malformation with significant improvement in their respiratory conditions. Two patients required additional subtotal surgical resection and one tracheostomy. Four patients remain under treatment. Toxicity was not observed. Conclusions: Sirolimus is a safe drug in neonates and can be considered the first therapeutical option in newborns at high risk of respiratory failure before sclerosis or surgery. Close follow-up is mandatory to identify side effects at long-term use.

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Section: Sirolimus In Neonates 507mentioning

confidence: 99%

Oral Sirolimus: An Option in the Management of Neonates with Life-Threatening Upper Airway Lymphatic Malformations

Triana

Miguel

Díaz

et al. 2019

Lymphatic Research and Biology

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“…Sirolimus is mainly distributed within red blood cells and has a large volume of distribution, being eliminated via fecal/biliary pathways with a half‐life of 62 hours. This long half‐life allows for convenient once‐daily dosing, but a loading dose is recommended to achieve target drug concentrations rapidly . Owing to its pharmacodynamics, it is essential that serum levels be monitored particularly in neonates in whom the drug‐metabolizing enzyme systems are in constant development and are still poorly understood …”

Section: Discussionmentioning

confidence: 99%

Preventive treatment with oral sirolimus and aspirin in a newborn with severe Sturge‐Weber syndrome

Junco

Sánchez‐Carpintero

López‐Gutiérrez

2019

Pediatric Dermatology

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Sturge-Weber syndrome (SWS) is characterized by facial capillary malformation, leptomeningeal capillary malformations, and choroidal and episcleral vascular malformations. These malformations produce neurologic and ophthalmological symptoms including seizures and glaucoma. A premature male newborn without prenatal diagnosis presented with severe bilateral SWS and was started on systemic sirolimus and aspirin. The patient has remained seizure-free for 23 months and demonstrated an excellent response to pulsed dye laser treatment. K E Y W O R D S laser, neonatal, therapy-systemic, vascular malformation F I G U R E 3 Patient at 4, 15, and 18 mo of age after sirolimus treatment and multiple PDL sessions | 527 Pediatric Dermatology TRIANA JUNCO eT Al.

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“…Genetic polymorphisms may impact the pharmacokinetics, i.e. absorption, distribution, metabolism and elimination of these agents (162,163).…”

Section: Pharmacogeneticsmentioning

confidence: 99%

Review of Genetic Variation as a Predictive Biomarker for Chronic Graft-Versus-Host-Disease After Allogeneic Stem Cell Transplantation

et al. 2020

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Pharmacokinetics, Pharmacodynamics, and Pharmacogenomics of Immunosuppressants in Allogeneic Hematopoietic Cell Transplantation: Part II

Cited by 33 publications

References 217 publications

Oral Sirolimus: An Option in the Management of Neonates with Life-Threatening Upper Airway Lymphatic Malformations

Oral Sirolimus: An Option in the Management of Neonates with Life-Threatening Upper Airway Lymphatic Malformations

Preventive treatment with oral sirolimus and aspirin in a newborn with severe Sturge‐Weber syndrome

Review of Genetic Variation as a Predictive Biomarker for Chronic Graft-Versus-Host-Disease After Allogeneic Stem Cell Transplantation

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