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IJESR Machine Learning Models for Assessment of HIV Biomarkers in Medicine,

ABSTRACT: Biomarkers have gained immense scientific and clinical value and interest in the
practise  of medicine.  Biomarkers  are potentially useful  in  diagnosis,  screening,  staging  and
selection of initial therapy. Advances in genomics, proteomics, and emerging high-throughput
technologies in medical practice are important sources to develop drugs for HIV/AIDS. 

In this paper  assessment of  HIV biomarkers  is studied  by  machine  learning  techniques  as it  is the important step to find out the better biomarker for HIV diagnosis and treatment. In future, these
novel approaches have provided opportunities to develop personalized treatment strategies for
HIV/AIDS.

Keywords: Biomarker, Diagnosis, High-throughput, Treatment, Personalized,

INTRODUCTION ;

 In 2001, a consensus panel at the „National Institutes of Health” defined the term biomarker as
„a characteristic that is objectively measured and evaluated as an indicator of normal biological
processes,  pathogenic  processes, or  pharmacologic responses  to a  therapeutic  intervention  or
other health  care intervention‟.  The  biomarker  is either produced by the diseased organ (e.g.,
tumour) or by the body in response to disease. Biomarkers are potentially useful along the whole
spectrum of the disease process. Before diagnosis, markers could be used for screening and risk
assessment. During diagnosis, markers  can determine staging, grading, and  selection of initial
therapy.  Later,  they  can  be  used  to  monitor  therapy,  select  additional  therapy,  or  monitor
recurrent diseases [1]. Thus, identifying biomarkers include all diagnostic tests, imaging technologies, and any other objective
measures of a person‟s health status. Hence biomarkers are also used in diagnosis of HIV-AIDS
[12], the world most deadly disease. AIDS is caused by HIV leading to severe immune damage
to human body and even death  at later stage. Potential of biomarkers lead to sufficient use in
AIDS for diagnosis, monitoring and selection of therapy. 

Biomarkers can also be used to reduce the time factor and cost for phase I and II of clinical trials
by replacing clinical endpoints.    Biomarkers span a broad sector of human health care and have
been around since the understanding of HIV-AIDS biology and other diseases to evolve.

Phases of evaluation of biomarkers
A clinical trial design may be  consist of following  phases which was  guided by the  National
Cancer Institute‟s “Early Detection Research Network” [2].

Phase  I:  It  refers to  preclinical  exploratory  studies.  Biomarkers  are  discovered  through
knowledge-based gene selection,  gene expression  or protein  profiling to  distinguish HIV  and
normal  samples.  Identified  markers  are  prioritized  based  on  their
diagnostic/prognostic/therapeutic  (predictive)  value  that  could  suggest  their  evolution  into
routine clinical use. The analysis of this phase is usually characterized by ranking and selection,
or finding suitable ways to combine biomarkers. It is preferred that the specimen for this phase of discovery comes from well-characterized cohorts, or from a trial with active follow-ups.
Phase  II: It  has  two  important  components.  Upon  successful  completion  of  phase  I
requirements, an assay is established with a clear intended clinical use. The clinical assay could
be a protein, RNA, DNA or a cell-based technique, including ELISA, protein profiles from MS,
phenotypic  expression  profiles,  gene  arrays,  antibody  arrays  or  quantitative  PCR.  The
significance of these techniques depends upon  two  parameters: firstly, such assays need  to be
validated for reproducibility and shown to be portable among different laboratories. Secondly,
the  assays  should  be  evaluated  for  their  clinical  performance  in  terms  of  „sensitivity‟  and
„specificity‟ with thresholds determined by the intended clinical use. 

Phase III:   During this phase, an investigator evaluates the sensitivity and specificity of the test
for the detection of diseases that have yet to be detected clinically. The specimens analyzed in
this evaluation phase are taken from study subjects before the onset of clinical symptoms, with
active  follow-up  of  HIV-AIDS  occurrence.  It  is  usually  time-  consuming  and  expensive  to
collect these samples with high quality; therefore, phase III should consist of large cohort studies
or intervention trials whenever possible. This is probably when the bio-marker will be ready for
clinical use. 

Phase IV: It evaluates the sensitivity and specificity of the test on a prospective cohort. A posi-
tive test triggers a definitive diagnostic procedure, often invasive and that could lead to increased
economic healthcare burden.  Therefore, in a phase IV study, an investigator can estimate the
false referral rate based on tested biomarkers and describe the extent and characteristics of the
disease detected (e.g., the  stage of HIV-AIDS at the time of detection). Sometimes, phase IV
requires a large cohort with long-term follow-up.

Phase V: In this phase, the overall benefits and risks of the new diagnostic test is evaluated. 

 Table 1. Performance characteristics of Biomarkers.
Response of biomarker
Disease present
Disease absent
Biomarker positive
A
B
Biomarker negative
C
D
D
AC
iseaseprevalence A B C D


  

1sensitivity
Negativelikelihoodratio specificity





D
Negativepredictivevalue CD



If the biomarker used as a diagnostic test, it should be sensitive and specific and have a high
predictive value as shown in table 1. A highly sensitive test will be positive in nearly all patients
with the HIV infection, but it may also be positive in many patients without the HIV infection.
To be of clinical value, most patients without the HIV infection should have negative test results. 

Characteristics of an ideal biomarker and basic statistical methods for evaluation
 An ideal biomarker should be safe and easy to measure.
 The cost of follow-up tests using biomarkers should be relatively low.
 It should be consistent across genders and ethnic groups.


Diagnostic odds ratio (DOR) of a biomarker represents the comprehensive ability of the marker:
11
sensitivity
specificity
DOR sensitivity specificity



Information  about  the  diagnostic  test  itself  can  be  summarized  using  a  measure  called  the
likelihood ratio. The likelihood ratio combines information about the sensitivity and specificity.
It tells how much a positive or negative result changes the likelihood that a patient would have
the disease. 

The likelihood ratio for a positive result (LR+) tells how much the odds of the disease increase
when a test is positive. The likelihood ratio for a negative result (LR−) tells how much the odds
of  the disease decrease  when a  test is  negative. The  likelihood ratio  can be  combined with
information  about  the  prevalence  of  the  disease,  characteristics  of  your  patient  pool,  and
information about a particular patient to determine the post-test odds of disease. To quantify the
effect of a diagnostic test, information about the patient is needed first. The pre-test odds, such as
the likelihood that the patient would have a specific disease prior to testing should be specified.
The pre-test odds are usually related to the prevalence of the disease, though it might be adjusted
upwards  or  down-wards  depending  on  characteristics  of  the  over-all  patient  pool  or  of  the
individual patient. Once pre-test odds have been specified, they are multi-plied by the likelihood
ratio to give the post-test odds:

post pre
odds odds likelihoodratio


The post-test odds represent the chances that a particular patient has a disease. It incorporates
information about the prevalence of the disease, the patient pool, and specific patient risk factors
(pre-test  odds)  and information  about the  diagnostic test  itself (the  likelihood ratio).     Most
biological markers, however, are not simply present or absent but have wide ranges of values
that  overlap  in persons  with  a  disease and  in  those  without it.  The  risk  typically  increases
progressively with increasing levels; few markers have a threshold at which the risk suddenly
rises, so various cut-off points must be evaluated for their ability to detect disease. Cut-off points
with high sensitivity, producing few false negative results, are used when the consequences of
missing a potential case  are severe, whereas  highly  specific  cut-off points, producing few false 

 positive  results,  are used  to avoid  mislabelling a  person who  is actually  free of  the  disease.
Sensitivity  and  specificity  calculated  at  various  cut-off  points  generate  a  receiver-operating-
characteristic  (ROC)  curve,  which  ideally  will  be  highly  sensitive  throughout  the  range  of
specificity. The most useful clinical tests are typically those with the largest area under the ROC
curve.    The use of multiple tests may also be considered

for screening. When multiple tests are obtained in series and the disease is  considered present
when  all  tests  are  positive  („AND  rule‟),  specificity  is  enhanced  whereas  sensitivity  is
diminished. When  multiple tests  are obtained  in parallel  and the  disease is  considered to  be
present  when  any of  the  tests  are  positive  („OR  rule‟),  sensitivity is  enhanced  and  specificity
diminishes [3]. 


Specific ways for Biomarker assessment:
1. Model discrimination: The C-statistic  or area under the receiver operating characteristic curve
(AUC) or ROC (receiver operating curve) is a popular method to test model discrimination. C-
statistic  for  a  multivariable  model  reflects  the  probability  of  concordance  among  persons  who
can be compared for a given outcome of interest and represents the probability that a case has a
higher or risk score (or a shorter time to event in survival analyses) than a comparable control.
The C-statistic  measures the concordance of the  score and disease state. The value of the C-
statistic ranges from  0.5 (no discrimination) to  1.0(perfect discrimination).  When considering
the efficacy of  novel bio-markers  in  risk  stratification,  one  approach  is  to  determine  to  what
extent  entering  the  can-didate  biomarker  into  standard  risk  prediction  models  will  actually
increase the model‟s C-statistic.

One  can grouping  of new  attributes  with  existing ones  (also  be  grouped  according  to same
characteristics). Machine learning is a subfield of computer science that evolved from the study
of pattern recognition and  computational learning theory in artificial  intelligence shows better
results in classification. Machine learning explores the study and construction of algorithms that
can  learn  from  and  make  predictions  on  data.  There  are  many  machine-  learning  software
available for analysis of data [5] i.e. WEKA software package [4]. Following are some of the methods which are inbuilt in WEKA and are good for analysis of data. 

(a)Decision tree (or tree diagram) is a decision support tool that uses a tree-like graph or model
of decisions and their possible consequences, including chance event outcomes, resource costs,
and utility. Decision  trees are commonly  used  in  operations research, specifically in decision
analysis, to help identify a strategy most likely to reach a goal. Another use of decision trees is as
a  descriptive  means  for  calculating  conditional  probabilities.  In  data  mining  and  machine
learning, a decision tree is a predictive model; that is, a mapping from observations about an item
to  conclusions  about  its  target  value.  More  descriptive  names  for  such  tree  models  are
classification  tree  (discrete  outcome)  or  regression  tree  (continuous  outcome).  In  these  tree
structures, leaves represent  classifications and branches represent conjunctions of features that
lead to those classifications. The machine learning technique for inducing a decision tree from
data is called decision tree learning, or (colloquially) decision trees [5].

(b)Naïve Bayes Classifier: - A naive Bayes classifier is a simple probabilistic classifier based
on applying Bayes' theorem with strong (naive) independence assumptions. A more descriptive
term  for the  underlying  probability  model would  be  "independent feature  model".  In simple
terms, a naive Bayes classifier assumes that the  presence (or lack of presence) of a particular
feature  of  a  class  is  unrelated  to  the  presence  (or  lack  of  presence)  of  any  other  feature
Depending on the precise nature of the probability model, naive Bayes classifiers can be trained
very  efficiently  in  a  supervised  learning  setting.  In  many  practical  applications,  parameter
estimation for naive Bayes models uses the method of maximum likelihood [6]. 
Of the various biomarkers described in literature CD4+, P24, IL10, IFN-γ can be as biomarkers
for prediction and diagnosis of HIV [12]. 

There are several limitations  to using increments  in  the C-statistic to determine the utility of
biomarkers in risk prediction [7]. First, the C-statistic depends to a large extent on the magnitude
of the association between a dichotomous exposure and outcome. Other limitations of C-statistic
include  low sensitivity  for determining  the  relative  importance of  different  risk factors  in a
multivariate model.

             IJESR        Volume 4, Issue 6        ISSN: 2347-6532
__________________________________________________________ 
A Monthly Double-Blind Peer Reviewed Refereed Open Access International e-Journal - Included in the International Serial Directories
Indexed & Listed at: Ulrich's Periodicals Directory ©, U.S.A., Open J-Gage as well as in Cabe ll’s Directories of Publishing Opportunities, U.S.A.
International Journal of Engineering & Scientific Research
http://www.ijmra.us

8
June
2016
2. Model calibration: A complementary step when analyzing the efficacy of a biomarker is to assess
the degree to which the biomarker improves model calibration.  This can be thought of as the
extent to which the expected risk (estimated by statistical models) agrees with the observed (or
true)  risk.  This  concept  may  be  important  when  counselling  patients  with  regards  to  their
numeric risk or probability of developing a given condition. A simple statistical test to compare
model discrimination with and without the biomarker of interest would fail to provide valuable
information  regard-ing  which  specific  groups  (i.e.,  which  deciles  or  quintiles  and  so on)  of
observed and expected risk are better explained by including a biomarker of interest.

3. Risk reclassification: The utility of a biomarker may also be assessed by studying how biomarker
information may lead to a reclassification of individuals in low medium- and high-risk categories
based on traditional risk factors. The ultimate goal of this approach is to refine risk stratification,
and  it  has been  particularly  emphasized when  considering biomarker  information that  would
serve to  shift individuals  who are in  the  intermediate-risk groups,  upwards into  the  high-risk
category or downwards into the low-risk category. Recent guidelines have recommended that the
individuals in the intermediate-risk category be targeted to undergo screening for existing HIV
[8].


4. Model validation: Models can be validated by 5-fold cross validation or 10 fold cross validation.
Boot strapping is one of the good methods in machine learning for validating model.

Considering multiple biomarkers for HIV:  Combinations of biomarkers with their accuracies as
obtained by machine learning techniques proves which combination is better for identification,
and diagnosis of HIV [12]

In  near  future if  any of the  HIV biomarker  is  identified, the  combinations  of biomarkers  is
improved and better one is again analysed by above discussed methods.

High throughput technologies for HIV biomarkers: These technologies are useful to assess
genomic  data  which  define  the  messages  and  the  resulting  protein  sequences  using  single
             IJESR        Volume 4, Issue 6        ISSN: 2347-6532
__________________________________________________________ 
A Monthly Double-Blind Peer Reviewed Refereed Open Access International e-Journal - Included in the International Serial Directories
Indexed & Listed at: Ulrich's Periodicals Directory ©, U.S.A., Open J-Gage as well as in Cabe ll’s Directories of Publishing Opportunities, U.S.A.
International Journal of Engineering & Scientific Research
http://www.ijmra.us

9
June
2016
nucleotide polymorphisms and different types of repeats. Transcriptomic data reveal the levels of
messages present. The basic idea in transcription profiling is to measure Mrna expression levels
of  thousands  of  genes  simultaneously  in  a  cell  or  tissue  sample  under  specific  conditions.
Proteomics  could  be  described  as  a  large  scale  study  of  protein  structure,  expression,  and
function (including modifications and interactions). Metabolomics is a whole cell measurement
of all the metabolites and it is considered to be equivalent to transcriptomics in Mrna expression
analysis. 

The  reason  for  using  high-throughput  technologies  is  that  they  provide  a  large  number  of
correlative data on gene or protein expression in relation to disease. Such data are then analyzed
for their association to  the disease.  The assumption  is that  multiple  variables will be  able to
provide information on associations more accurately than a single variable (marker). Such strong
associations provide  major impetus for the molecular profiling approaches to find patterns or
profiles for a clinical test based on high dimensional gene or protein expression panels [9]. Comparative  genomic  analyses  have  yielded  a  large  number of  genomic  expression data  in
relation to disease. The patterns of gene expressions that are observed, represent novel signatures
for  the  respective diseases  and  can  be used  to  develop  new  clinical tests  based  upon  gene
expression patterns, and identify candidate markers for diagnosis and prognosis. 

Single  nucleotide polymorphisms  have also  been  used as  genetic  markers of  risk,  treatment
response,  and  gene  and  environment  interactions.  These  high-throughput  technologies  have
significantly increased the number of potential DNA, RNA, Protein biomarkers under study. One
of the major problems with  high-dimensional data derived from high-throughput genomic and
proteomic  technologies  is  overfitting  of  the  data  when  there  are  large  numbers  of  potential
predictors  among a  small  number of  outcome  events.  For  example,  a  recent study  of  RNA
microarray analysis showed  how  easy it  was  to overfit  data  with a  small  number of  samples.
Simon and colleagues clearly demonstrated that expression data on 6000 genes from imaginary
individuals, 10 normal and 10 cases, could be used to discover discriminatory patterns, using one
common  method,  with  98% accuracy  [10].  Many  of  the  so-called  „omics‟  derived  data  are
subjected to a similar over-fitting if the training and validation sets for analyses are small and not
             IJESR        Volume 4, Issue 6        ISSN: 2347-6532
__________________________________________________________ 
A Monthly Double-Blind Peer Reviewed Refereed Open Access International e-Journal - Included in the International Serial Directories
Indexed & Listed at: Ulrich's Periodicals Directory ©, U.S.A., Open J-Gage as well as in Cabe ll’s Directories of Publishing Opportunities, U.S.A.
International Journal of Engineering & Scientific Research
http://www.ijmra.us

10
June
2016
randomized.  Most  commonly  used  approaches  to  analyze  „omics‟  data  are  artificial  neural
networks, boosted decision tree analyses, various types of genetic algorithms and support vector
machine-learning algorithms. Each approach has the potential to over fit the data. Over fitting has
led to  strong  conclusions  that  are  likely  to  be  erroneous.  The  first  step,  therefore,  would  be  to
determine whether  the results are reproducible and  portable. For this  purpose, information on
samples should be blinded and samples be sent to several laboratories for running the sample sets
under a fixed protocol. The data from each laboratory should be analyzed by an independent data
manager to learn if each laboratory reproduced a similar result. Splitting the samples randomly
between „training  sets  and  validation sets‟  should  minimize the over  fitting. The validation set
should not contain samples used in training sets ANTIRETROVIRAL THERAPY & HIV BIOMARKERS

CD4+ and viral load are used as biomarkers for diagnosis and starting treatment for HIV.

Table 2: Relation between CD4+cells,viral load and stages of HIV.
CD4+T cells
Greater than 500 per micro litre of blood
Stage I
Viral load is low. 
Less than 500 per micro litre of blood
Stage II
Less than 350 per micro litre of blood
Stage III
Viral  load
reaches  to
millions.
Less than 200 per micro litre of blood
Stage IV

The level of virus in the body continues to rise and CD4+T cell count continues to fall. Some
illnesses that develop in people infected with HIV leads to the need for antiretroviral therapy.
The illnesses include HIV-related kidney diseases and certain opportunistic infections. ART is a
lifelong treatment that helps people  with HIV live  longer and healthier lives. Available drugs
called Highly  active anti-retro viral  therapy  (HAART). HAART  provides effective  treatment
options  for  treatment-naive  and  treatment-experienced  patients.  Six  classes  of  antiretroviral
agents currently exist, as follows:
 Non-nucleoside reverse transcriptase inhibitors (NNRTIs) 
 Protease inhibitors (PIs) 
 Integrase inhibitors (INSTIs) 
 Fusion inhibitors (FIs) 
 Chemokine receptor antagonists (CCR5 antagonists) 
Each class targets a different step in the viral life cycle as the virus infects a CD4+ T lymphocyte
or other target cell. The use of these agents in clinical practice is largely dictated by their ease or
complexity of use, side-effect profile,  efficacy based on clinical evidence, practice guidelines,
and clinician preference.

Resistance, adverse effects,  pregnancy, and  confections with  hepatitis B  virus, or  hepatitis C
virus  present  important  challenges  to  clinicians  when  selecting  and  maintaining  therapy  for
HIV/AIDS. Combination antiretroviral therapy (cART) has significantly reduced morbidity and
mortality of HIV-infected patients, yet their life expectancy remains reduced compared with the
general population. Most HIV-infected patients receiving cART have some persistent immune
dysfunction characterized  by chronic  immune activation  and premature  aging of  the immune
system.  Biomarkers of T-cell activation (CD69, -25 and -38, HLA-DR, and soluble CD26 and -
30)  is  reviewed;  generalized  immune  activation  (C-reactive  protein,  IL-6  and  D-dimer);
microbial translocation (lipopolysaccharide, 16S rDNA, lipopolysaccharide-binding protein and
soluble CD14); and immune dysfunction of specific cellular subsets (T cells, natural killer cells
and  monocytes) in  HIV-infected  patients on  cART and  their relationship  to adverse  clinical
outcomes including impaired CD4 T-cell recovery, as well as non-AIDS clinical events, such as
cardiovascular disease are studied [13]. Drug development based on molecular biomarkers and targeted personalised medicine for
HIV 
In the treatment of diseases especially AIDS, there is a shift from the traditional clinical practices
to novel approaches. Traditionally HIV positive patients are treated with nucleotide, nucleoside,
protease,  integrase  inhibitors. However,  recent  advances  in basic  and  clinical  research have
provided  opportunities  to develop  „personalized‟ treatment  strategies.  These novel  approaches
             IJESR        Volume 4, Issue 6        ISSN: 2347-6532
__________________________________________________________ 
A Monthly Double-Blind Peer Reviewed Refereed Open Access International e-Journal - Included in the International Serial Directories
Indexed & Listed at: Ulrich's Periodicals Directory ©, U.S.A., Open J-Gage as well as in Cabe ll’s Directories of Publishing Opportunities, U.S.A.
International Journal of Engineering & Scientific Research
http://www.ijmra.us

12
June
2016
are intended to identify individualized patient benefits of therapies, minimize the risk of toxicity
and reduce the cost of treatment. The biggest challenge for researchers and clinicians today is, to
decide on which type of biomarker to use across the wide spectrum of disease processes. 

The evolving trend is the usage of patterns of markers instead of a single marker. This approach
could, to some extent, reduce the error rate in predicting the outcome or severity of side effects
during  the  targeted  therapies.      With  the  increasing  knowledge  of  the  molecular  pathways
underlying the development of various  diseases, the selection of patients and their efficacy in
future will be based  on  molecular  profiling  or phenotypic expression of their target molecules.
These targeted drugs shut down their specific pathway or sets of pathways. The predictability of
the response to targeted drugs rules out their use in all patients, which helps to avoid unnecessary
drug-associated side effects.

CONCLUSION AND FURTHER SCOPE: Machine learning techniques are better to identify
the  correct  biomarker or  combination  of biomarkers  for prediction,  diagnosis, prognosis  and
treatment monitoring  of diseases.  A large concerted  effort  is required  to  advance  the  field  of
biomarker discovery. Most current biomarkers do not satisfy the required characteristics for use
among  the  spectrum of  diseases.  Validation  of  new biomarkers  is  necessary.  Generation of
prospective data  will be  necessary  for  validation  and demonstration  of clinical  utility. High-
throughput technologies  have  begun  to  define disease processes and other biological processes
with  molecular biology  detail  and thus  offer  the potential  to  identify and  characterize novel
biomarkers. Molecular biology is now seen as encouraging more „personalized medicine‟ – the
closer  alignment  of biological information  (derived from  molecular diagnostics)  and therapy
selection.  Well  designed  efforts  will  be  needed  to  develop  general  knowledge  about  the
molecular history of diseases, to keep up with the progress with biomarkers development. The
evolution of molecular  medicine, coupled  with the  discovery and  clinical application  of new
biomarkers, will  play  a  significant  role  in  reshaping  medicine  as  a  science.       Science  in  India
could make a significant impact on the global scene if scientists and policy makers could agree to
dedicate sufficient time  and  resources  to  the  field  of biomarkers. This should be much beyond
task-force and excellence initiatives, and should be output-driven in a defined time line.
   
DR, ANUBHA DUBEY PHD BIOINFORMATIC
   RESEARCHER AND CONSULTANT
  

 

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