8.4.0.0 Di­ag­no­sis of Mono­genic Di­a­betes

A di­ag­no­sis of one of the three most com­mon forms of MODY, in­clud­ing GCK-‍MODY, HNF1A-‍MODY, and HNF4A-‍MODY, al­lows for more cost-‍ef­fec­tive ther­a­py (no ther­a­py for GCK-‍MODY; sul­fony­lureas as first-‍line ther­a­py for HNF1A-‍MODY and HNF4A-‍MODY). Ad­di­tion­ally, di­ag­no­sis can lead to iden­tification of other af­fect­ed fam­i­ly mem­bers.

A di­ag­no­sis of MODY should be con­sid­ered in in­di­vid­u­als who have atyp­i­cal di­a­betes and mul­ti­ple fam­i­ly mem­bers with di­a­betes not char­ac­ter­is­tic of type 1 or type 2 di­a­betes, al­though ad­mit­ted­ly “atyp­i­cal di­a­betes” is be­com­ing in­creas­ingly difficult to pre­cise­ly define in the ab­sence of a defini­tive set of tests for ei­ther type of di­a­betes. In most cases, the pres­ence of au­toan­ti­bod­ies for type 1 di­a­betes pre­cludes fur­ther test­ing for mono­genic di­a­betes, but the pres­ence of au­toan­ti­bod­ies in pa­tients with mono­genic di­a­betes has been re­port­ed (121). In­di­vid­u­als in whom mono­genic di­a­betes is sus­pect­ed should be re­ferred to a spe­cial­ist for fur­ther eval­u­a­tion if avail­able, and con­sul­ta­tion is avail­able from sev­er­al cen­ters. Read­i­ly avail­able com­mer­cial ge­net­ic test­ing fol­low­ing the cri­te­ria list­ed below now en­ables a cost-‍ef­fec­tive (122), often cost-‍sav­ing, ge­net­ic di­ag­no­sis that is in­creas­ingly sup­ported by health in­sur­ance. A biomark­er screen­ing path­way such as the com­bi­na­tion of uri­nary C-‍pep­tide/​cre­a­ti­nine ratio and an­ti­body screen­ing may aid in de­ter­min­ing who should get ge­net­ic test­ing for MODY (123). It is crit­i­cal to cor­rect­ly di­ag­nose one of the mono­genic forms of di­a­betes be­cause these pa­tients may be incor­rect­ly di­ag­nosed with type 1 or type 2 di­a­betes, lead­ing to subop­ti­mal, even po­ten­tially harm­ful, treat­ment reg­i­mens and de­lays in di­ag­nos­ing other fam­i­ly mem­bers (124). The cor­rect di­ag­no­sis is es­pe­cial­ly crit­i­cal for those with GCK-‍MODY mu­ta­tions where mul­ti­ple stud­ies have shown that no com­pli­ca­tions ensue in the ab­sence of glu­cose-‍lowering ther­a­py (125). Ge­net­ic coun­sel­ing is rec­om­mend­ed to en­sure that af­fect­ed in­di­vid­u­als un­der­stand the pat­terns of in­her­i­tance and the im­por­tance of a cor­rect di­ag­no­sis.

The di­ag­no­sis of mono­genic di­a­betes should be con­sid­ered in chil­dren and adults di­ag­nosed with di­a­betes in early adult­hood with the fol­low­ing find­ings:

Di­a­betes di­ag­nosed with­in the first 6 months of life (with oc­ca­sion­al cases pre­senting later, most­ly INS and ABCC8 mu­ta­tions) (120,126)

Di­a­betes with­out typ­i­cal fea­tures of type 1 or type 2 di­a­betes (neg­a­tive di­a­betes-‍as­so­ci­at­ed au­toan­ti­bod­ies, nonobese, lack­ing other metabol­ic fea­tures es­pe­cial­ly with strong fam­i­ly his­to­ry of di­a­betes)

Sta­ble, mild fast­ing hy­per­glycemia (100–150 mg/dL [5.5–8.5 mmol/‍L]), sta­ble A1C be­tween 5.6 and 7.6% (be­tween 38 and 60 mmol/‍mol), es­pe­cial­ly if nonobese