Palifermin Compared to Supersaturated Calcium Phosphate in Prevention of Oral Mucositis after Stem Cell Transplantation Open Access

Hadid, Tarik (Summer 2018)

Permanent URL: https://etd.library.emory.edu/concern/etds/5m60qr98k?locale=en
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Abstract

Oral mucositis (OM) is a common, debilitating complication of conditioning regimens for hematopoietic stem cell transplantation (HSCT). Supersaturated calcium phosphate rinse (SCPR) and palifermin have shown efficacy in preventing OM. However, whether their efficacy differs is unknown.

This study compares SCPR to palifermin in HSCT patients receiving radiotherapy-based myeloablative conditioning. A comprehensive review of our institutional database was performed to identify patients who received myeloablative conditioning therapy between 2008 and 2012. The majority of patients received Fludarabine, Busulfan and total body irradiation.

A total of 26 patients received SCPR and 122 patients received palifermin for OM prophylaxis. Both groups were monitored for development of OM. Multivariable logistic regression analysis was employed to estimate the adjusted odds ratio (aOR) comparing the odds of developing OM between groups and adjusting for age, gender, primary diagnosis, conditioning regimen, donor source and disease status. The odds of developing World Health Organization (WHO) grade 3 or 4 OM were significantly lower in the palifermin group (57% vs 100%, aOR=0.03, p=0.01). Moreover, the palifermin group had lower WHO grade 4 OM (22% vs 62%, aOR=0.19, p=0.0006). The overall odds of developing OM of any grade were not significantly different between the two groups (86% for palifermin group vs 100% for SCPR group, aOR=0.14, p=0.15). Subgroup analyses demonstrated the superiority of palifermin in preventing severe OM, regardless of age, sex, primary diagnosis, donor source, and disease status.

In conclusion, retrospective data suggest that palifermin was more effective than SCPR in preventing severe grades of OM in HSCT patients receiving radiotherapy-based myeloablative conditioning.

Table of Contents

CHAPTER .............................................................................................................................. PAGE

 

TITLE........ ..................................................................................................................................... .i

 

DISTRIBUTION AGREEMENT .................................................................................................. .ii

 

APPROVAL SHEET ..................................................................................................................... iii

 

ABSTRACT COVER SHEET ....................................................................................................... iv

 

ABSTRACT .....................................................................................................................................v

 

COVER SHEET............................................................................................................................ .vi

 

ACKNOWLEDGMENTS ............................................................................................................ vii

 

TABLE OF CONTENTS ............................................................................................................. viii

 

INTRODUCTION AND LITERATURE REVIEW .......................................................................1

 

 

Hematopoietic stem transplantation .....................................................................................1

 

Conditioning regimens ...............................................................................................1

 

The process of HSCT .................................................................................................2

 

Types of HSCT ..........................................................................................................2

 

Indications for HSCT .................................................................................................3

 

Complications of HSCT .............................................................................................3

 

Mucositis ..............................................................................................................................4

 

Definition and clinical implications ...........................................................................4

 

 

Incidence of OM and its risk factors ........................................................................4

 

Stages of OM .............................................................................................................5 ix

 

Pathobiology of OM ..................................................................................................6

 

Prevention of OM ......................................................................................................6

 

Treatment of OM .......................................................................................................8

 

Problem statement .................................................................................................................10

 

Purpose statement .................................................................................................................10

 

Research questions ................................................................................................................11

 

 

 

Significance...........................................................................................................................11

 

 

Journal selection....................................................................................................................12

 

METHODS AND MATERIALS ...................................................................................................12

 

 

Study type ..........................................................................................................................12

 

Study participants...............................................................................................................12

 

Sample.....................................................................................................................12

 

Inclusion criteria .....................................................................................................13

 

Exclusion criteria ....................................................................................................13

 

HSCT Process ....................................................................................................................13

 

Coding of HSCT .....................................................................................................13

 

 

Conditioning regimens ..........................................................................................13

 

Study drugs .........................................................................................................................14

 

Outcomes ............................................................................................................................15

 

Assessment of outcomes .........................................................................................15

 

Statistical analysis ...............................................................................................................16 x

 

Ethical considerations .........................................................................................................17

 

 

 

RESULTS ......................................................................................................................................17

 

 

Efficacy ...............................................................................................................................19

 

Distribution of OM .............................................................................................................22

 

Prediction of OM ................................................................................................................22

 

Subgroup analysis ...............................................................................................................23

 

DISCUSSION ................................................................................................................................25

 

 

Limitation ..........................................................................................................................28

 

Implication for public health ..............................................................................................29

 

Conclusions and future directions ......................................................................................29

 

REFERENCES ..............................................................................................................................30

 

APPENDIX ....................................................................................................................................39

 

FIGURES AND TABLES .............................................................................................................18

 

 

Figure 1 ..............................................................................................................................18

 

Figure 2 ..............................................................................................................................22

 

Figure 3a ............................................................................................................................24

 

Figure 3b ............................................................................................................................25

 

Table 1 ...............................................................................................................................19

 

Table 2a ..............................................................................................................................21

 

Table 2b .............................................................................................................................21

 

Table 3 ...............................................................................................................................23

 

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