Treatment and Prognosis of Immune (Idiopathic) Thrombocytopenic Purpura in Children

7/26/2019 Treatment and Prognosis of Immune (Idiopathic) Thrombocytopenic Purpura in Children

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Treatment and prognosis of immune (idiopathic)thrombocytopenic purpura in children

All topics are updated as new evidence becomes available and our peer reviewprocess is complete.Literature review current through: Mar 2013. | This topic last updated: Apr ! 2011.

INTRODUCTION " #mmune $idiopathic% thromboc&topenic purpura $#T'% o(

childhood is characteri)ed b& ac*uired thromboc&topenia and is a generall& benign

disorder o( un+nown cause. #T' is also re(erred to as autoimmune

thromboc&topenic purpura or isoimmune thromboc&topenic purpura.

Although both acute and chronic (orms o( #T' e,ist! the& have similar presentation

in a child who presents with #T'. Twent& percent o( a((ected children will go on to

have chronic #T'! generall& de(ined as persistent thromboc&topenia (or more than

si, months be&ond presentation. -owever! some authors have suggested that 12

months is a more appropriate parameter (or de(ining chronic #T'.

The treatment and prognosis o( acute and chronic #T' in children will be reviewed

here. The epidemiolog&! clinical mani(estations! and diagnosis o( #T' in children are

discussed separatel&.

OVRVI! " The management o( children with #T' is the subect o( much debate

because it is unclear whether /watch(ul waiting/ or pharmacologic intervention

provides the most appropriate care (or a((ected children.

'roponents o( /watch(ul waiting/ without pharmacologic intervention base their

decisions upon the (ollowing observations:

event& to 0 percent o( children with #T' will recover within a (ew monthso( presentation with or without treatment.

Most patients do not have serious bleeding including those with platelet

counts 10!000microL. #ntracranial hemorrhage! the most seriousconse*uence o( #T'! is rare with an incidence o( 0.1 to 0. percent.

#n one report (rom the #ntercontinental 4hildhood #T' tud& 5roup! the ris+

o( severe bleeding (ollowing diagnosis was 0.6 percent in patients with no!mild! or moderated bleeding and was unrelated to initial management.

Those who (avor pharmacologic intervention base their decision upon the evidencethat pharmacologic therapies are available that raise the platelet count more rapidl&

than i( no treatment was administered. The& suggest drug treatment be used in

patients with serious bleeding or who are at ris+ (or serious bleeding. #n a

retrospective stud& (rom (ive 7ordic countries $weden! 8inland! 9enmar+! 7orwa&!

and #celand%! earl& treatment accelerated platelet recover& in patients with acute

#T'! but did not reduce the ris+ o( developing chronic #T' or morbidit&.

There are no randomi)ed trials comparing pharmacologic therap& and observation

upon the outcome o( children with #T'. 9ata based upon retrospective studies

suggest that pharmacologic therap& does not reduce the ris+ o( developing chronic

#T' but it remains unclear whether pharmacologic therap& reduces the ris+ o( li(e
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threatening complications associated with acute #T' because o( their low prevalence

rate.

;oth the American ociet& o( -ematolog& $A-% and ;ritish ociet& o( -aematolog&

have published guidelines (or the management o( #T' in children and adults. ;oth

societies ac+nowledge that published evidence (or recommendations is wea+. As aresult! both sets o( guidelines are based primaril& upon e,pert opinions and

observational studies.

The A- guidelines $published in 166


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'resence o( severe or li(ethreatening bleeding

>is+ o( signi(icant bleeding! such as a child undergoing a procedure that is

li+el& to induce blood loss! or a child with a plateletcount10!000microL and signs o( cutaneous bleeding $bruising!petechiae! andor purpura%

An& concomitant or pree,isting condition that increases the ris+ o(

thromboc&topenia or bleeding $eg! hemophilia%

?hen it is decided to use pharmacologic therap&! treatment options include

corticosteroids! intravenous immunoglobulin $#@#5 or #5#@%! or intravenous anti

>ho$9% immune globulin. everal studies have shown that the duration o(

s&mptomatic thromboc&topenia is shortened b& an& o( these three modalities

compared to no treatment $table 1A4%.

A stud& (rom the #ntercontinental 4hildhood #T' tud& $#4#% registr&! which

included selected patients (rom 36 countries and 1B institutions voluntaril&

reported b& ph&sicians! demonstrated the variabilit& o( management (or childhood

#T'. >eview o( the treatment data! which were available (or 16B< patients! showed

that therap& included corticosteroids alone $33 percent%! #@#5 alone $26 percent%! a

combination o( corticosteroids and #@#5 $B percent%! and no pharmacologic

intervention $31 percent%.

Corticosteroids" 4orticosteroids have been used (or man& &ears (or the

management o( #T' in all age groups. teroids are presumed to reduce the ris+ o(

s&mptoms in #T' patients b&:

>educing antibod& production

>educing reticuloendothelial s&stem phagoc&tosis o( antibod&coated

platelets

#mproving vascular integrit&

#mproving platelet production

#n randomi)ed trials o( children with #T'! prednisone therap& has been shown to

more rapidl& increase platelet counts than placebo. #n one stud&! children treated

with oral prednisone $2 mg+g per da&% (or 1 da&s (ollowed b& a taper over a wee+

had a higher platelet count and lower bleeding scores compared to those who

received placebo. #n another stud&! children treated with oral prednisone

$2 mg+g per da&% (or 21 da&s were more li+el& to have a platelet count greater

than 30!000microL with the (irst 10 da&s o( treatment compared to placebo

treated children $60 versus percent%.

A variet& o( dose regimens have been used ranging (rom prednisone at a dose o(

2 mg+g per da& (or two to (our wee+s! to high pulse doses o( intravenous or

oral meth&lprednisolone $0 mg+g per da&% (or three to seven da&s $table 1A4%.

ome evidence suggests that time o( response and rate o( response are dose

related! but comparative studies among the multiple regimens are (ew and involve

onl& small numbers o( patients. Thus! there is insu((icient evidence to recommend
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two treatment modalities were made at 2 hours $>> 0.> 0.3! 6D 4# 0.Bho$9% immunoglobulin Fanti>ho$9% #gG in patients with #T' who

were >ho$9%positive.

These trials demonstrated that anti>ho$9% #g is e((ective in improving plateletcounts. Limited data comparing the e((icac& o( anti>ho$9% #g versus #@#5 and

corticosteroids include the (ollowing two studies:

The (irst stud& was a randomi)ed controlled trial in which 1< patients were

assigned to receive one o( (our treatmentsE #@#5 $1000 mg+g (or twodoses on consecutive da&s%! #@#5 as a single dose $00 mg+g%! anti>ho$9% #g $2 microgram+g (or two doses on consecutive da&s%! or oralprednisone $initial dose mg+g tapered and weaned over 21 da&s%. At B2

hours a(ter the start o( treatment! the groups treated with #@#5 had thebest response to therap&. >esponse to treatment! as de(ined b& the

percent o( patients with a posttreatment platelet count greater than

20!000! was seen in 6B! 6! 2! and B6 percent o( patients treated withone dose o( #@#5 $00 mg+g%! two doses o( #@#5 $each 1000 mg+g%! two
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doses o( Anti>ho$9% #g $each 2 micrograms+g%! and oral prednisone!respectivel&.

-emoglobin levels (ell below 10 gdL in 2 percent o( patients treated withanti>ho$9% #g. 'atients who received #@#5 were more li+el& to have

complaints o( (ever! nausea! vomiting! and headache.

The second stud& was a randomi)ed controlled trial in which 10 patients

were assigned to receive #@#5 as a single dose o( 00 mg+g! or anti>ho$9% #g as a single dose o( either B microgram+g or

0 microgram+g. At 2 hours a(ter the start o( therap&! the response totherap& with a platelet count greater than 20!000 was BB! 0! and B2percent in patients treated with #@#5! anti>ho$9% #g$0 microgram+g%! and anti>ho$9% #g $B microgram+g%! respectivel&.

;& da& seven! hemoglobin levels had decreased b& 0.3! 1.ho$9% #g $0 microgram+g%! and anti>ho$9% #g$Bmicrogram+g% groups! respectivel&. The side e((ects o( headache!

(ever! or chills occurred in all three groups but were less (re*uent in thegroup treated with the lower dose o( anti>ho$9% #g.

Thus! anti>ho$9% #g is e((ective in the treatment o( children with #T' and with

higher doses appears to have comparable results to #@#5. -owever! anti>ho$9% #g

is associated with an increased incidence o( signi(icant hemol&sis. imilarl&! in a

retrospective report! patients treated with anti>ho$9% #g e,perienced an increased

rate o( adverse e((ects as compared with those treated with #@#5.

;ased upon these results! in patients with #T' when earlier recover& o( platelet

numbers is desirable! we pre(er to treat with #@#5 rather than anti>ho$9% #g. ?e

administer #@#5 as a single dose o( 1000 mg+g.

#( anti>ho$9% is used! it should onl& be given to children with a hemoglobin level

greater than 10 gdL. 'atients also should be screened with a 4oombs antibod& to

identi(& the rare patient with Hvans s&ndrome. #n addition! care(ul monitoring (or

acute hemol&sis andor hemoglobinuria! and their possible se*uelae should be

per(ormed.

Our approach" #( pharmacologic therap& is used! we pre(er #@#5 to

corticosteroids or intravenous anti>ho$9% #g because it reliabl& increases platelet

counts and more *uic+l& than corticosteroids! and is sa(er than anti>ho$9% #5. ?e

administer #@#5 as a single 1000 mg+g dose. Alternate options include intravenous

or oral corticosteroids! di((erent dosing regimens o( #@#5! and intravenous anti

>ho$9% #g.

latelet transfusions" The onl& indication (or platelet trans(usions is li(e

threatening hemorrhage! such as intracranial hemorrhage. Largerthannormal

doses are re*uired because normal doses are ine((ective due to platelet destruction.

$onitoring " Almost all children with #T' are managed in the ambulator& setting.

'atients who receive pharmacologic intervention are usuall& hospitali)ed (or an

average length o( sta& o( two da&s.

#n the ambulator& setting! platelet counts are generall& monitored once or twice

wee+l&! depending on the clinical situation and severit& o( thromboc&topenia. ?hen

recover& o( platelet counts is detected! the interval between measuring platelet


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counts is lengthened! but monitoring should continue until the platelet count has

returned to normal $I10!000microL% and is stable. This return occurs within one

month a(ter presentation in onehal( o( children with #T'! and b& three months in

about B0 percent o( children.

#n our practice! we stop monitoring a(ter the platelet count has returned to normaland has remained stable (or two to three months. >ecurrence o( acute #T' is rare!

probabl& occurring in less than 1 percent o( cases! although the possibilit& o( well

compensated chronic #T' cannot be ruled out.

ince all #T' therapies are tempori)ing interventions intended to reverse

e,peditiousl& a real or perceived ris+ (or hemorrhage! the& do not need to be

continued until normal platelet counts are reached. Therap& is targeted to increase

the platelet count above a threshold $usuall& greater than20!000microL% that stops

bleeding or eliminates the ris+ o( serious bleeding.

#ifethreatening bleeding" Li(ethreatening bleeding is rare. Although

intracranial hemorrhage $#4-% has an incidence o( onl& 0.1 to 0. percent! it is themost serious conse*uence o( #T'. The presence o( signi(icant headache should

prompt care(ul evaluation (or an& other neurologic signs! and earl& diagnostic

imaging should be considered to identi(& #4-.

#( #4- or an& other li(ethreatening hemorrhage occurs! immediate intervention

should be given to the a((ected patient. Cur practice is consistent with the

recommendations o( the previousl& mentioned 2010 #nternational consensus report

and includes the (ollowing:

'latelet trans(usions

#@#5 " 1000 mg+g per da& (or two da&s

Meth&lprednisolone " 30 mg+g per da& administered intravenousl& (or

three da&s

8or #T' patients with unstable or progressive #4-! emergenc& craniotom& ma& be

necessar&. Hmergenc& splenectom& ma& be considered in selected clinical situations

but onl& as a last resort.

C*RONIC IT " Appro,imatel& 20 to 30 percent o( children who present with #T'

will have chronic #T'! de(ined as persistent thromboc&topenia $platelet count lessthan 10!000microL% be&ond si, months (rom the time o( presentation. 'atients

with chronic #T' are usuall& clinicall& indistinguishable (rom those with acute #T' at

presentation.

#ndividuals with chronic #T' should undergo evaluation to e,clude other causes o(

thromboc&topenia! such as chronic in(ections $including -#@%! bone marrow (ailure!

collagen vascular disorders! and other autoimmune or immunode(icienc& disorders.

tudies should include viral antibod& titers! bone marrow e,amination! and studies

(or collagen vascular disorders $eg! antinuclear antibod& test%.

$anagement " #n up to onethird o( children with chronic #T'! spontaneous

remission will occur months or even &ears later. 4hildren &ounger than 10 &ears o(

age are more li+el& to remit than older patients and treatment is usuall& not
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necessar&. 4hildren older than 10 &ears o( age! especiall& adolescent (emales! have

a disease course more li+e that seen in adults with #T' and should be treated in a

similar manner.

#n chronic #T'! platelet counts tend to range between 20!000

and B!000microLE conse*uentl&! man& patients will re*uire no treatment. #t isver& uncommon (or an individual with chronic #T' to have a platelet count less

than 10!000microl.

Management o( children with chronic #T' should (ocus on minimi)ing the individual=s

ris+ (or bleeding. 'harmacologic intervention is used as a tempori)ing measure. #n

some cases! such as patients with chronic severe #T'! splenectom& is per(ormed

because o( persistent signi(icant hemorrhagic s&mptoms that re*uire repeated!

sometimes almost continuous! pharmacologic interventions.

harmacologic therapy" #n children with chronic #T'! pharmacologic therap&

usuall& is used when patients have signi(icant bleeding! or re*uire surger& or dental

e,traction. Adolescent girls ma& have e,cessive bleeding during their menses andma& re*uire therapeutic intervention. Therap& generall& raises the platelet count

temporaril&.

'harmacologic options are similar to those used in the initial management o(

patients with #T' at presentation and include the (ollowing:

4orticosteroids " 'atients ma& receive periodic short courses or pulses o(

corticosteroids. 8or patients who are steroid dependent in order to remains&mptom (ree! alternate da& dosing ma& be e((ective in preventingbleeding while reducing side e((ects. 'rolonged dail& steroid treatment isto be avoided because o( its adverse e((ects! especiall& poor growth. #n

our practice! we pre(er to avoid the prolonged use o( glucocorticoidtherap&.

#mmunoglobulin therap& " #ntravenous #g5 $#@#5% or anti>ho$9% also have

been e((ective in chronic #T' patients! even those who are resistant to

steroids. All o( these strategies o((er temporar& alleviation o( s&mptomsand improvement in platelet numbers! but no data demonstrate an e((ecton resolution o( chronic #T'.

#n our practice! #@#5 is administered at a dose o( 00 mg+g per da& (or two da&s

and repeated when s&mptoms recur.

&plenectomy " A small percentage o( patients with chronic #T' will have

persistent signi(icant hemorrhagic s&mptoms and re*uire repeated! sometimes

almost continuous! pharmacologic interventions. 8or such patients! the ris+s and

bene(its o( splenectom& must be considered.

plenectom& is e((ective in improving the platelet count and reducing the

associated ris+ o( bleeding in


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The anticipated improvement in hemostasis and platelet count must be balanced

with the small ris+ o( overwhelming postsplenectom& in(ection! which ma& be li(e

threatening. ;ecause o( the high in(ection rates in &ounger asplenic individuals!

tempori)ing therapies are especiall& important (or patients &ounger than to

&ears o( age with chronic #T'. 'resplenectom& immuni)ations and subse*uent

penicillin proph&la,is are necessar& (or all age groups.

'latelet counts in splenectomi)ed patients generall& are (ollowed (or an inde(inite

periodE an& decompensation in platelet counts or increase in s&mptoms should

prompt assessment (or the presence o( an accessor& spleen.

Treatment of refractory chronic IT " Appro,imatel& 2 to 30 percent o(

children with chronic #T' have ongoing hemorrhagic problems a(ter splenectom&. #n

these cases! evaluation should identi(& an& possible accessor& spleen! which should

be removed! i( present.

>itu,imab is a chimeric murinehuman anti4920 monoclonal antibod& which

targets autoantibod& producing l&mphoc&tes. #t promises to be use(ul in treatingboth primar& and secondar& re(ractor& chronic #T' in children with reported

remission rates o( about 30 percent.

#n an open label stud& o( 3< children $2 to 16 &ears o( age% in the Jnited

tates who received a wee+l& intravenous doseo( ritu,imab $3Bmgm2% (or (our doses! ritu,imab improved the platelet

count to greater than 0!000microL in 11 children $31 percent%. -owever!si, patients had serious side e((ects including two with serum sic+ness. #na one&ear (ollowup stud&! eight o( the 11 initial responders maintained aplatelet count greater than 10!000microL without (urther interventions.Cne initiall& nonresponding patient achieved remission a(ter 1< wee+s andtwo additional patients maintained platelet counts o( 0!000microL.

#n another stud& o( 6 #talian children $2 to 1 &ears o( age% with re(ractor&

#T' treated with ritu,imab $3B mgm2 wee+l& (or (our wee+s%! 3patients responded $


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#n the original cohort o( 01 patients (rom the 7ordic ociet& (or 'ediatric

-aematolog& and Cncolog& $7'-C% stud&! 2 patients $B percent% received

pharmacologic treatment within two wee+s o( diagnosisE in most cases one or two

doses o( #@#5 were administered. i,month (ollowup data were available (or 06

patients. The (ollowing (indings were noted:

C( the 06 patients! 3B< children had a platelet count less

than 20!000microL during the course o( their disease and were classi(iedas being at ris+ (or serious bleeding.

#n the highris+ group o( patients! the ris+ period (or serious bleeding lasted

less than one month in B percent o( patients and persisted (or more than

si, months in 10 percent.

There were 11 events that re*uired medical attention during the si,month

(ollowup period. More than hal( o( the events $B reports% wereaccounted (or b& the 101 patients with chronic #T'.

Most o( the reported events were due to cutaneous bleeding $bruising!

petechiae! andor purpura%. There were 33 episodes o( mucosal bleeding!which included 23 reports o( epista,is! < reports o( oral bleeding! 1 reporto( menorrhagia! and 1 report o( rectal bleeding.

7one o( the events were li(ethreatening and there were no reports o(

intracranial hemorrhage or signi(icant trauma. #n 1 cases! blood

trans(usions were re*uired.

#n the 06 patients with complete (ollowup data! there was no di((erence in

the ris+ o( developing chronic #T' in patients who did not receivetreatment compared to those who did $23 versus 2< percent%.

#n the #ntercontinental 4hildhood #T' tud& $#4#% stud&! three o( the 1B2 patients

with available si,month (ollowup data had intracranial hemorrhage. The (ollowing

(indings were noted:

#nitial platelet counts (or the three patients were 000! 11!000!

and 1

Management varied with one patient receiving no pharmacologic therap&!

one treated with corticosteroids! and one received a combination o(corticosteroids and #@#5.

ubse*uent (ollowup data were onl& available (or two o( the three patients.

The patient who received both corticosteroids and #@#5 died within the(irst wee+ o( diagnosis o( #T' and the patient who received corticosteroidalone (ull& recovered without neurologic se*uelae.

A small number o( children with acute #T' $ percent%! who initiall& remit with

recover& o( a normal platelet count! will have an episode o( recurrent acute

thromboc&topenia. ?hen it occurs! it usuall& is a sel(limited event and

management principles remain the same as (or the initial acute episode. 'rolonged

monitoring o( such patients is necessar& to eliminate the possibilit& that chronic #T'

is the actual diagnosis.


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IN+OR$"TION +OR "TINT&" JpTo9ate o((ers two t&pes o( patient education

materials! KThe ;asics and K;e&ond the ;asics. The ;asics patient education

pieces are written in plain language! at the th to < th grade reading level! and the&

answer the (our or (ive +e& *uestions a patient might have about a given condition.

These articles are best (or patients who want a general overview and who pre(er

short! eas&toread materials. ;e&ond the ;asics patient education pieces arelonger! more sophisticated! and more detailed. These articles are written at the

10 th to 12 th grade reading level and are best (or patients who want indepth

in(ormation and are com(ortable with some medical argon.

-ere are the patient education articles that are relevant to this topic. ?e encourage

&ou to print or email these topics to &our patients. $ou can also locate patient

education articles on a variet& o( subects b& searching on Kpatient in(o and the

+e&word$s% o( interest.%

&U$$"R, "ND RCO$$ND"TION&" #mmune $idiopathic% thromboc&topenic

purpura $#T'% o( childhood is characteri)ed b& thromboc&topenia $platelet countusuall& less than 20!000microL% that is an ac*uired and usuall& benign disorder.

#n children with #T'! the ris+ o( serious bleeding is small regardless o( whether

pharmacologic treatment is administered.

Initial medical management

The management o( childhood #T' is the subect o( much debate because it

is unclear whether observation alone or observation with pharmacologicintervention in de(ined circumstances provides the most appropriate care(or the child with #T'.

?hether or not pharmacologic therap& is used! ph&sical activit&! especiall&

contact sports! should be restricted! and medications with eitherantiplatelet or anticoagulant activit& should be avoided.

Cptions (or pharmacologic intervention include corticosteroids! intravenous

immunoglobulin $#@#5%! or intravenous anti>ho$9% immune globulin

Fanti>ho$9% #gG. These agents raise the platelet count more *uic+l& thanno therap&.

8or children with #T' with severe or li(ethreatening bleeding! we

recommend pharmacologic therap& in $ 5rade 1; %. 8or these patients! we

also recommend platelet trans(usion $ 5rade 14 %. #n our practice! weadminister platelets! highdose glucocorticoids! and #@#5 to children withli(ethreatening bleeding.

8or children with #T' with a platelet count less

than 10!000microL with cutaneous bleeding $bruising!petechiae andor purpura%! or (or those with anticipated ris+ (or signi(icant

bleeding $eg! patient scheduled (or surger& or dental e,traction%! wesuggest pharmacologic therap& $ 5rade 24%. ?e also suggest

pharmacologic therap& in patients who have a concomitant or pree,istingcondition that increases the ris+ o( thromboc&topenia or bleeding $eg!hemophilia% $ 5rade 24 %.
https://4aa595ceecaf9011bca85edb079a7f1b880b53ef.googledrive.com/host/0B6A3S-PEqav2QzhrVTlFQmJzaGs/contents/._grade_2?title=Grade%201Bhttps://4aa595ceecaf9011bca85edb079a7f1b880b53ef.googledrive.com/host/0B6A3S-PEqav2QzhrVTlFQmJzaGs/contents/._grade_3?title=Grade%201Chttps://4aa595ceecaf9011bca85edb079a7f1b880b53ef.googledrive.com/host/0B6A3S-PEqav2QzhrVTlFQmJzaGs/contents/._grade_6?title=Grade%202Chttps://4aa595ceecaf9011bca85edb079a7f1b880b53ef.googledrive.com/host/0B6A3S-PEqav2QzhrVTlFQmJzaGs/contents/._grade_6?title=Grade%202Chttps://4aa595ceecaf9011bca85edb079a7f1b880b53ef.googledrive.com/host/0B6A3S-PEqav2QzhrVTlFQmJzaGs/contents/._grade_2?title=Grade%201Bhttps://4aa595ceecaf9011bca85edb079a7f1b880b53ef.googledrive.com/host/0B6A3S-PEqav2QzhrVTlFQmJzaGs/contents/._grade_3?title=Grade%201Chttps://4aa595ceecaf9011bca85edb079a7f1b880b53ef.googledrive.com/host/0B6A3S-PEqav2QzhrVTlFQmJzaGs/contents/._grade_6?title=Grade%202Chttps://4aa595ceecaf9011bca85edb079a7f1b880b53ef.googledrive.com/host/0B6A3S-PEqav2QzhrVTlFQmJzaGs/contents/._grade_6?title=Grade%202C


12/14

#( treatment is indicated! we suggest administering #@#5 rather than

corticosteroids or intravenous anti>ho$9% #g $ 5rade 2; %. #n our practice!we administer #@#5 as a single 1000 mg+g dose. Alternate optionsinclude intravenous or oral corticosteroids! di((erent dosing regimens o(#@#5! and intravenous anti>ho$9% #g.

Chronic IT " About 20 to 30 percent o( children with #T' will have chronic #T'!

de(ined as persistent thromboc&topenia $platelet count less

than10!000microL% si, months a(ter presentation.

The management (or childhood chronic #T' includes the (ollowing:

Hvaluation to e,clude other disorders.

#n children &ounger than 10 &ears o( age! treatment is usuall& unnecessar&

because the& are li+el& to have spontaneous remission. ?e recommendthat pharmacologic therap& be used as a temporar& measure during

episodes o( signi(icant bleeding! or (or surger& or dental e,traction$ 5rade 1;%.

4hildren with chronic #T' who are older than 10 &ears o( age! especiall&

adolescent (emales! have a disorder that is more li+e that seen in adultswith #T' and should be treated in a similar manner.

plenectom& is reserved (or patients who continue to have signi(icant and

persistent hemorrhagic s&mptoms re*uiring repeated pharmacologicinterventions 12 months a(ter diagnosis.

-. &teroids in childhood IT I2. N2013 JpTo9ate O

/. Clinical trial e'idence0 effecti'eness of

glucocorticoids1 IVIg1 and antiD in initial treatmentof IT in childrenI

"uthors&tudyN

op.

age

+ollo2up

Randomi3edtreatment arms

Outcome measurelateletcount

4leedingsymptom

s

"d'erseffects

Deaths

Mc?illiamsand Mauner

2B < &rmean

7> 'rednisone $2mg+gd , 21 d%

Median time to platelet counto( 10P

21 d 7> 7> 0

7o treatment $p Q.03% 7> 0

artorius 63 umpelLeedetest

'rednisoneI 'lacebo$p .01%

'rednisone placebo$b&>umpelLeede test

7> 0

'lacebo 7> 0

;uchananand-olt+amp

2B 11&r

2 d 'rednisone $2mg+gd , 1 d! thentaper to d 21%

'latelet count! ;leeding time!clinical bleeding score at d 02

'rednisoneI placebo$p .0%onl& at d B

'rednisone placebo$p .0%onl& at d B$bleedingtime andclinicalscore%

#ncreasedappetite!weight gain

0

'lacebo 0

#mbach et al 6 1 7> 7>

. 7>: not reported.5. Reproduced with permission from George, JN, Woolf, SH, Raskob, G, et al.

!lood "##$% &&'(. )op*right + "##$ merican Societ* of Hematolog*.

5. Clinical trial e'idence0 effecti'eness of

glucocorticoids1 IVIg1 and antiD in initial treatmentof IT in childrenII (continued)

"uthors&tudy

Nop.age

+ollo2up

Randomi3ed treatmentarms

Outcome measurelateletcount

4leedingsymptoms

"d'erseffects

Deaths

;elluci etal

1 7> 0

'rednisone $1 mg+gd , 3w+%

BB percent$no di((erence%

7> 7> 0

Phali(a etal

30 2 mo1&r

I< mo M=prednisolone $#@! 10mg+gd , d%

Mean platelet count onda&s 11

7> 7> 0

'rednisone $2 mg+gd ,

w+%

7> 7> 0

#@#g $0. g+gd , d% 7> 7> 0

C)so&le etal

20 2 mo11&r

I< mo M=prednisolone $po! 30mg+gd , 3 d! then 20mg+gd , d%

'roportion with plateletcount I10P in 3 d! 0

;lanchetteet al

3 B mo1 &r

10 d 'rednisone $ mg+gd , Bd! then tapered to d 21% #@#g$1 g+gd , 2 d%

Median time to plateletcount I20P! I0P

2 d! d 7> ?eight gain!behavioralchange Bpercent withnausea!vomiting!headache! (ever

0

7o therap& 1 d! 2 d $p .01 vs

prednisone% d! 1< d $p0.1 vs eithertreatment%

0

;lanchette 1< < mo1&r


14/14

#@#gregimens%

10 gdL

B. 7>: not reported.&. Reproduced with permission from George, JN, Woolf, SH, Raskob, G, et al.


6. Clinical trial e'idence0 effecti'eness ofglucocorticoids1 IVIg1 and antiD in initial treatmentof IT in childrenIII (continued)

"uthors

&tudy N

op.age

+ollo2up

Randomi3edtreatment arms

Outcomemeasure

lateletcount

4leedingsymptoms

"d'erseffects

Deaths

Alba&ra

+ et al

B 2 mo

1B &r

< mo M=prednisolone

$po! 30 mg+gd ,

Bd%

Mean platelet

count on

da&s 030

Mean

I100P b& d

. 7o

di((erence

among

groups

7> #ncreased

appetite and

4ushingoid

appearance

0

M=prednisolone$po! 0 mg+gd ,

B d%

1 #4- #ncreasedappetite and

4ushingoidappearance

0

#@#g $0. g+gd ,

d%

7> 1 pt with

asepticmeningitisE 2

withheadache!

vomiting

0

10.7>: not reportedE #4-: intracerebral hemorrhage."".Reproduced with permission from George, JN, Woolf, SH, Raskob, G, et al.


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Treatment and Prognosis of Immune (Idiopathic) Thrombocytopenic Purpura in Children