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Cerebral rhizomucor infection treated by posaconazole delayed release tablets in an allogeneic stem cell transplant recipient

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Cerebral Rhizomucor Infection Treated by Posaconazole Delayed Release Tablets in an Allogeneic Stem Cell Transplant Recipient 12 3 4 5 Q1 6 7 Q2 8 9 10 11 12 Q4 13 14 15 16 17 18 19 20 21 22 23 24 25[.]

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1 Case Report

5 Q1Diego O Andreya,* , Laurent Kaisera, Stephan Emoneta, Veronique Erardb,

10

11 1 Introduction

12 Q4 Rhizomucor is an emerging fungal pathogen in allogeneic

13 hematopoieticstemcelltransplant(allo-HSCT)recipients

belong-14 ingtothegroupof Zygomycetes.1 Itsincidenceis rising in the

15 contextofwidevoriconazoleuse.CerebralRhizomucorinfectionis

16 associated with especially high mortality and treatment must

17 combineaggressivesurgicaldebridement,antifungaltherapyand

18 restoration ofimmune function wheneverpossible.2–4 Recently

19 posaconazole delayed-release tablets, registered for antifungal

20 prophylaxis,haveallowedyieldinghigherplasmalevelsthanthe

21 conventionalposaconazoleoralsolution.5–7Neverthelesstherole

22 of posaconazole new formulations remains to be defined in

23 therapeuticindications.Herewedescribethecaseofa cerebral

24 Rhizomucor infection successfully treated by posaconazole

25 delayed-releasetabletsinanallo-HSCTrecipient

26

2 Casereport

27

A30-year-oldBrazilianwomendiagnosedwithacute

myelo-28 monocyticleukemiaunderwentcytarabine-basedchemotherapy

29

At admission she presented with several pulmonary nodular

30 lesions on CT-scan, suggestive of fungal origin The initial

31 treatment consisted in empirical voriconazole (Vfend1, Pfizer)

32

200mgevery12hoursi.v.duringthreeweeks.Duetoradiological

33 progressiondespiteantifungaltherapysheunderwentasurgical

34 resection consisting in a right inferior lobectomy and wedge

35 resections of two left superior lobe nodules Microbiological

36 analysisofthenodulesrevealedpositivedirect examinationfor

37 mycelium elements identified as Rhizomucor pusillus by 18S

38 panfungalqPCRfollowedbysequence-basedidentification

(Gen-39 BankaccessionnumberHQ845298.1),whereascultureremained

40 negative Liposomal amphotericin-B (AmBisome1, Gilead

41 Sciences)at5mg/kg/dayfollowedbyposaconazoleoral

suspen-42 sion(Noxafil1,MSD)400mgevery6hours,wereadministeredfor

43 sixmonthsandsubsequentlystoppedduetoremission

44 Nineteenmonthsaftertheinitialdiagnosisleukemiarelapsed

45 and the patient underwent FLAG (fludarabine, idarubicine,

46 cytarabine, granulocyte colony stimulating factor (G-CSF))

che-47 motherapy.Atadmissionanewpulmonaryexcavatedlesionwas

International Journal of Infectious Diseases xxx (2016) xxx–xxx

Keywords:

Zygomycosis

Mucormycosis

Mucormycosis(zygomycosis)isanemergingfungaldiseaseinallogeneichematopoieticstemcell transplant (allo-HSCT) recipients A 30-year-old woman diagnosed with acutemyelomonocytic leukemiaand needingallo-HSCT presentedpulmonary andcerebralinfectionduetoRhizomucor pusillus.Thisfungalinfectionwastreatedwithsurgicaltreatmentandposaconazoledelayed-release tablets This strategy allowed reaching high drug levels that could not be obtained with the posaconazolesolution

ß2016TheAuthor(s).PublishedbyElsevierLtdonbehalfofInternationalSocietyforInfectiousDiseases ThisisanopenaccessarticleundertheCCBY-NC-NDlicense(

http://creativecommons.org/licenses/by-nc-nd/4.0/)

Q3

ContentslistsavailableatScienceDirect

j o urn a l hom e pa ge : ww w e l s e v i e r c om/ l o ca t e / i j i d

http://dx.doi.org/10.1016/j.ijid.2016.12.014

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48 seen in the left superior lobe (at the sites of prior wedge

49 resections).Aleftsuperiorlobectomywasperformed.Pathological

50 and microbiological examinations, as well as panfungal PCR

51 (fungal inter spacer region (ITS) conventional PCR) remained

52 negative.8 Suspecting a relapse of the Rhizomucor infection,

53 liposomal amphotericin-B was restarted at 5mg/kg/day The

54 patientremainedinaplasia,and allogeneichematopoieticstem

55 celltransplantation(allo-HSCT)wasconsideredtheonlylifesaving

56 option In the pre-transplantation workup performed under

57 meropenem and liposomal amphotericin-B treatment, three

58 cerebralnoduleswerefoundintherightmiddletemporalgyrus,

59 therightfrontalinferiorgyrusandtherightfrontalinferiorgyrus

60 oncerebralMRI,motivatingadditionofposaconazolesuspension

61 CSFculturesandpanfungalPCR,bloodcultures,aswellasblood

62 galactomannanes remained negative After two inconclusive

63 cerebral biopsy attempts, a surgical resection of the most

64 peripheral cerebral nodule was performed Direct examination

65 (fungifluor) was positive for mycelium elements, with large

66 septationsandabsenceofbranches,(seeFig.1 confirmingthe

67 suspicionofacerebralRhizomucorinfection,probablysecondaryto

68 ahematogeneousdisseminationfromthelungduringtheinitial

69 episode,containedduringtheremissionperiod,andsubsequently

70 relapsingduringaplasia.Liposomalamphotericin-Bwasincreased

71 to 10mg/kg/day and oral posaconazole was switched from

72 solution800mg/daytodelayed-releasetablets300mg/day,due

73 tolow plasmaposaconazole(0.22mg/L).On thesameday

allo-74 HSCT(HLAhaplo-identical,donatedfromhersister,nonTdepleted

75 with myeloablative conditioning regimen including

cyclophos-76 phamide,fludarabineandi.v.busulfanandtheuseofhighdoses

77 cyclophosphamidepost-transplantationasgraft-versushost

pro-78 phylaxis)wasperformed.Duetolowposaconazoleplasmablood

79 levels (0.45mg/L), the dosing was increased to 400mg/day,

80 allowingtherapeuticlevelstobereached

81 Three weeks after resection of the cerebral fungal abscess

82 liposomalamphotericin-Bwas taperedto5mg/kg,followed by

83 3mg/kg, and finally discontinued after a total of two months

84 Posaconazoledelayedreleasetabletswerecontinuedwithplasma

85 druglevelssuperiorto2mg/L.Afterfourmonths,hematological

86 recoverywascompleteandthecerebrallesionshaddisappeared

87 onMRI,allowingreductionto300mg/day.Sixmonthspost

allo-88 HSCT, the patient was considered in remission from the

89 mucormycosis Unfortunately the patient died due toARDS in

90 thesettingofCMVpneumoniaandpulmonaryGVHD

91

3 Discussion

92

In hematological malignancies and bone marrow transplant

93 patients,lungandrhino-cerebral arethemostcommonsites of

94 Rhizomucorinfection.CNSabscessesinfectionsarenotrare;ina

95 largepublishedseriesofmucormycosis(zygomycosis),283cases

96 involved the brain.2 These infections are severe, of rising

97 importance and incidence probably in the context of wide

98 voriconazoleusefor both treatment ofinvasiveaspergillosis or

99 for secondaryprophylaxis.Cerebralmucormycosis isassociated

100 withahighmortalityrateof50%.3

101 Cerebralfungalinfections, andespecially cerebral

mucormy-102 cosis,remaindifficulttotreatbecauseofthepoorCNSpenetration

103

of most antifungals, as wellas theabsence of activity against

104 Zygomycetesof mostantifungals,includingfluconazole,

vorico-105 nazoleandechinocandins.Cureisrarelyachievedwithoutsurgical

106 removal

107 Itraconazole, posaconazole and the newly FDA-approved

108 isavuconazole have activity against Zygomycetes In salvage

109 therapies, posaconazole, which has moderate CNS penetration

110 appearedasacornerstonefortreatmentof(cerebral)

mucormy-111 cosis with a response rate of >60%.9–12 Although no exact

112 therapeuticguidelinesexist forcerebral mucormycosis,

posaco-113 nazolesolutionisrecommendedforsalvagetherapyataposology

114

of800mg/dayinadditiontohighdoseliposomal

amphotericin-115

B.13Howeverachievingadequateposaconazoleserumlevelshas

116 beenchallengingwiththeoralsolution.Inthis setting,

posaco-117 nazole delayed-released tablets, achieving higher serum levels

118 thantheoralsolutionareapromisingoption

119 HerewedescribeacaseofacerebralRhizomucorinfectioninan

120 allo-HSCT recipient treated successfully with an antifungal

121 regimen containing posaconazole delayed-release tablets and

122 liposomal amphotericin B, combined to surgery It is to our

123 knowledge the first description of such therapeutic option in

124 cerebral mucormycosis Posaconazole delayed-release tablets

125 allowed to reach therapeutic drug levels > 2mg/L that could

126 notbeobtainedwiththestandardsolution

127 Fewinformationareavailableforposaconazoledelayed-release

128 tablets,PhaseIstudiesshowed>97%reachingtherapeuticlevels

129 with300mgoncedailyinPK/PDstudies.14,15

130

No published clinical trials are available on efficacy of

131 posaconazoledelayed-releasetabletsinthepreventionofIFIsin

132 immunocompromised patients,althoughit seemsprobablethat

133 achievinghigherbloodposaconazolelevelwillconferatleastas

134 good protection against IFI than the suspension The place of

135 posaconazoledelayedtabletsforIFItreatmentneedstobedefined

136

asnoclinicaltrialsareavailable.Sofarthereisonlyasinglereport

137

onacaseofa65-year-oldmanwithanAspergillusbrainabscess,

138 treatedwithsurgicalresectionandvoriconazoleforoneyear,and

139 subsequentlytreatedwithposaconazoledelayed-releasetabletsat

140

300mg/dayefficientlyandsafely.16

141 Our observation suggests that posaconazole delayed-release

142 tabletsareaninterestingtherapeuticoptionforcerebral

mucor-143 mycosis,duetothehigherdruglevelsobtained.Thesemighteven

144

beincreasedwiththemorerecentlyFDA-approvedintravenous

145 posaconazoleformulation.Clinicaltrialswiththesenew

posaco-146 nazole formulations are needed and will hopefully show an

147 increaseofsuccessfulresponsetotreatmentandsurvival

148 Acknowledgments

149

We thank Dr Arnaud Riat from The HUG Microbiology

150 Laboratoryforprovidingthepicture

151 Fundings:Thisresearchdidnotreceiveanyspecificgrantfrom

152 funding agencies in the public, commercial, or not-for-profit

153 sectors

2

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154 Authorcontributions:DOADraftingarticle;LK,SE,VE,YC,CVD:

155 Criticalrevisionofarticle

156 Conflictsofinterest:None

157 References

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