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A clinical study evaluating the effect of eppikajutsuto on lymphatic malformations (LMEP)

A clinical study evaluating the effect of eppikajutsuto on lymphatic malformations (LMEP)

20

Nineteen children (mean age: 24.1 months [range, 0-85 months]; 10 males and 9 females) with macrocytic, microcystic, or mixed lymphatic malformations confirmed by magnetic resonance imaging were enrolled.

Patient enrollment and data collection/analysis of the primary and secondary endpoints have been completed. The manuscript is currently in preparation.

In this study, adverse events occurred in 3 out of 19 patients (15.8%), including grade 4 neutropenia, and grade 1 infection and hepatic dysfunction. One patient also experienced an upper respiratory tract infection and urticaria; however, all events were mild and did not require discontinuation of treatment.

Percentage of lesions with a >-20% reduction in volume was seen in 10 of the 19 patients, 0.5263 (90% CI: 0.3201-0.7261). A >-50% volume reduction was observed in 4 of the 19 patients (21.1% [90% CI: 7.5-41.9%]). Median percentage change in lesion volume was -22.9% (range, -142.3-96.0%). QOL improvement was assessed in only three patients aged >5 years. Among them, 2 (66.7%) patients achieved a >-10% improvement in QOL scores. Medication adherence of >-70% was observed in 17 of the 19 patients (89.5%).

Eppikajutsuto (EKJT) demonstrated promising efficacy in reducing LM volume, particularly in head and neck lesions, with a potentially favorable safety profile. Compared with sirolimus, EKJT may offer an alternative therapeutic approach with distinct mechanisms and tolerability advantages.

Mar. 31, 2026

No

none

https://jrct.mhlw.go.jp/latest-detail/jRCTs041210007

Ogawa Keiko

Hiroshima University hospital

1-2-3 Kasumi Minami-ku Hiroshima-city Hiroshima, Japan

okeiko22@hiroshima-u.ac.jp

Ogawa Keiko

Hiroshima University hospital

1-2-3 Kasumi Minami-ku Hiroshima-city Hiroshima, Japan

+81-82-257-1921

okeiko22@hiroshima-u.ac.jp

Complete

April. 16, 2021

Interventional

non-randomized controlled trial

open(masking not used)

active control

single assignment

treatment purpose

1. Diagunostic image (at least Echo,CT,MRI)demonstrates a specific LM lesion
2. Weight 25kg or less

When any one or more of the following items are satisfied
Has a history of allergic reactions to Eppikajutsuto.
Have received or have received mTOR inhibitors
Has undergone sclerotherapy / surgery in the past
Has a serious disease other than lymphatic malformation
Participated in other clinical studies (studies) within 3 months before the start of administration of the test drug
Evaluable MRI cannot be taken
When the principal investigator or the investigator determines that apatients is inappropriate for this study

Both

Lymphatic malformation

Patients take the test drug "Eppikajutsuto" for 6 months. The dose is adjusted according to the body weight of the study subject, and 0.6 g / min 3 per kg of body weight per day is orally administered before meals. The amount of oral medication should be increased or decreased as the body weight is increased or decreased. The upper limit of medication is 7.5 g / day.

The response of tumor volume due to volumetry of lymphatic malformation at 6 months after drug administration from before the start of administration, volume reduced by 20% at 6 months, and in the case of diffuse lesions, the reduction of major lesions are evaluated in the same manner.

1. Ratio of volume reduced by 50% or more
2. Reduction rate of lesion
3. 10% improvement of QOL with treatment
4. Safety of administration of Eppikajutsuto
5. Medication adherence of Eppikajutsuto, Ratio of people who could take 70
% or more

+81-76-265-2048

April. 13, 2021

none