Adrenocortical Carcinoma: Prognostic Indices Based on Clinical and Immunohistochemical Markers

Vaė Dichtchekenian1, Carlos Alberto de Bragança Pereira2. Hilton Kuperman1, Thais Della Manna’, Durval Damiani1, Venancio Avancini Ferreira Alves3, Adhemar Longato Filho3 and Nuvarte Setian1

1 Pediatric Endocrinology Unit, Instituto da Criança Hospital das Clínicas, São Paulo University School of Medicine, 2Institute of Mathematics and Statistics, University of São Paulo and 3 Department of Pathologic Anatomy, Hospital das Clinicas, São Paulo University School of Medicine, Brazil

ABSTRACT

Adrenocortical carcinoma is a rare condition with an unpredictable prognosis as a rule. The authors retrospectively analyzed the clinical outcome of 46 patients (31 F, 15 M) during 16 years building up a numerical index for the prognosis, based on clinical and immunohisto- chemical data. Four indices were analyzed:

J1 =

; Y + 2L + 4H T J2 = (J1)

W V 200 ;

W

J3 = O+Y+2L+4H T

; J4 = (J3)

V 200

Y = 1 when chronological age (CA) >33 mo, Y = 0 when CA <33 mo; L = 1 for right sided tumor and L = 0 for left sided tumor; H = 1 in presence of hypertension and H = 0 for normal blood pressure; T = length of disease in months; W = weight of tumor (g); O = 1 in the absence of p53 protein and O = 0 in the presence of p53. The chance of bad prognosis was observed when age is >33 mo, tumor is on the right side, systemic hypertension is present, tumor weight >250 g, in the absence of p53, J1, J2, J3 >0.4 (p <0.001) and J4 >0.5 (p <0.01). Clinical data and the mathe- matical model enabled us to establish prob- abilities of good prognosis in 78-96% and bad prognosis in 63-83%.

Reprint address: Vae Dichtchekenian Rua Canario 1331 CEP 04521-005 São Paulo, Brazil e-mail vae@uol.com.br

VOLUME 18, NO. 4, 2005

KEY WORDS

adrenocortical carcinoma, prognosis, mathematical model, children

INTRODUCTION

Adrenocortical carcinoma (AC) is rare in children and adolescents, having a variable and potentially fatal course characterized by elevated production of adrenal hormones. In the last 16 years we have registered 46 cases, while in other countries the numbers are not so striking1-6.

The diagnosis is usually made on clinical grounds, since the majority of AC in children express endocrine manifestations, such as Cushing’s syndrome, virilization, precocious puberty and systemic hypertension. Surgical removal of a localized tumor, in cases without metastatic disease, is the most effective form of therapy but does not determine the prognosis. Neither histo- logical studies, according to Medeiros and Weiss12, nor the quantification of the DNA content and tumor size, have proven to be a sufficient basis for establishing the prognosis of the AC5.7-13. With this aim, immunohistochemical and molecular studies have been suggested as the most appropriate tools to study the behavior of this type of tumor14-17

Immunohistochemical studies were undertaken with the objective of determining the biological behavior of the neoplastic cells, and in particular the proliferating cell nuclear antigen (PCNA), which could be a good marker of cellular multiplication giving a prognostic index18. The p53 gene has been the subject of various studies and it has been named the ‘guardian of the genome’, protecting DNA either by blocking the cell cycle or by inducing

apoptosis in disordered cells19-22. In special situa- tions, when a mutation occurs, the activity of the gene reverts to such an extent that its modified protein promotes cell transformation and tumori- genesis17-2 17-24

Based on the difficulty of the prognosis for AC in pediatric patients, we decided to use mathe- matical formulas to provide a numerical index derived from clinical and immunohistochemical data regarding to p53 protein, with the objective of determining a more reliable prognosis and hence a more appropriate therapeutic strategy25-27.

PATIENTS AND METHODS

Forty-six children and adolescents (31 girls and 15 boys) were enrolled in this retrospective study. The main complaints were the presence of viriliza- tion or precocious puberty. Twenty-eight were alive after 20 months to 16 years of follow up, and 18 had a malignant outcome: seven metastasized widely and 11 died. The characteristics of the patients are summarized in Table 1. The histo- logical study of 33 patients was based on the criteria of Medeiros and Weiss12, which take into account nine items: 1. high nuclear grade; 2. pre- sence of over five mitoses per 50 high-power fields; 3. atypical mitotic figures; 4. 75% or more of the cells with eosinophilic cytoplasms; 5. 33% or more of the tumoral architecture being diffuse; 6. the presence of necrosis; 7. tumoral invasion of the smooth musculature of the venous vascular wall; 8. sinusoidal invasion; and 9. capsular invasion. The presence of three or more of the above criteria suggests malignant tumoral behavior.

For the immunohistochemical study of the altered p53 protein, we used sections of paraffin- embedded blocks and DO-1 monoclonal antibodies (Santa Cruz Technology, USA) which are specific for paraffin-embedded samples. We considered the presence of specific staining either in the nucleus or in the cytoplasm as positive.

Data were statistically analyzed with respect to the following: age of the patient (Y); laterality of the tumor (L); presence of systemic hypertension (H); tumor weight (W); immunohistochemical aspects of the p53 protein (O); and delay from the onset of symptoms to the time of diagnosis (T).

We created four indices, J1, J2, J3 and J4 (see below), with the objective of establishing a single variable which contains all information, such as the patient’s age, laterality of the tumor, systemic hypertension, duration of disease, tumor size and immunohistochemical properties of the p53 protein. The variables placed in the numerator with a positive sign and multiplied by a given value are directly related to the development of the disease. The duration of disease variable is inversely related and placed as the denominator. To ensure an easily compared final value the square root divided by 200 is justified by the high numerical value of the weight (g). To demonstrate the close correlation between these indices and the development of the disease, we applied a Wilcoxon test24 to analyze the ‘benign course’ in opposition to the ‘malignant course’ groups. To evaluate the results, we cate- gorized the index and performed a x2 test. The median for J1, J2, and J3 is 0.4, while for J4 it is 0.5. We estimated the probabilities of the prognosis according to these indices.

The J1 index took into consideration the duration of the disease, T (months); age of the patient (when over 33 months Y = 1 and when less than or equal to 33 months Y = 0); site of the tumor, L = 1 (right) and L = 0 (left); and H = 1 or H = 0 if systemic hypertension is present or absent, respectively. With the 45 patients (excluding those for whom the tumor size was not known), we reached J2 where W is the tumor weight in grams. For the 33 patients who had their p53 gene analyzed (O), we also defined two indices: J3 for unknown tumor weight, and J4 (n = 32) for known tumor weight. If immunohistochemical tests are negative, O = 1; if positive, O = 0.

J1 = Y +2L+4H ; J2= J1 ; T V 200

J3 = 0+Y+2L+4H T ; J4 = J3. V 200 W

RESULTS

Although there was a clear predominance of females (n = 31, 67.4%) compared to males (n =

JOURNAL OF PEDIATRIC ENDOCRINOLOGY & METABOLISM

TABLE 1: Characteristics of the adrenocortical carcinoma in 46 patients
Patient #SexYLHTW0Course
1F24 / 001416000
2F50 / 110425011
3F33 / 000933010
4F9/00127011
5F24 / 0001285I0
6F13 / 000311000
7F77 / 111643011
8F120/ 11171,29511
9F66 / 11061,43001
10F22 / 0101500
11F66 / 110275001
12F39 / 1111877011
13F122/ 10161,10001
14F22 / 00196500
15F22 / 01133001
16F4/00128000
17F14/00033010
18F59 / 100301910
19F23 / 00155000
20F57 / 100305010
21F31/000245500
22F15/00175010
23F30/000121500
24M22 / 000122400
25M38 /110121,53011
26M32 / 001181500
27M68 /1112035001
28M33 / 010214500
29M100 / 100135011
30M19/0011212000
31M47 / 101180011
32M120 / 101170011
33M36 / 10053900
34F36 / 11012150
35F147 / 101126001
36F66 / 111223601
37F41 /10053820
38F69 / 100550
39F17/01066300
40F148 / 10121,3801
41F58 /10019100
42M14/0018100
43M67 / 10111,000I
44M4/01121500
45M106 / 11012150
46M39 / 1117800

Y: age (months). Age >33 months Y = 1; < 33 months Y = 0. L: side of tumor: right L = 1; left L = 0.

H: systemic hypertension: present H = 1; absent H = 0. T: duration of disease in months. W: tumor weight in grams.

O: p53 protein immunohistochemistry: negative O = 1; positive O = 0. Course: bad = 1; good = 0.

15, 32.6%), there was no correlation between the sex of the patients and a good or bad outcome (p = 0.60). The same occurred with the criteria of Medeiros and Weiss12 (p = 0.37).

We observed a higher probability of bad prognosis among patients aged over 33 months (p <0.01), duration of disease less than or equal to 7 months (p <0.05), tumor on the right side (p <0.05), presence of systemic hypertension (p <0.05), tumor weight greater than 250 g (p <0.001) and immunohistochemical test negative for p53 protein (p = 0.06). J1, J2 and J3 with values greater than 0.4 (p <0.01) and J4 greater than 0.5 (p <0.01) indicate bad prognosis (Table 2).

The calculation of the associated probability with the indices J1, J2, J3 and J4 showed a possibility of a good outcome in 78-96% of the patients and a bad one in 63-83% (Table 3).

DISCUSSION

All of our patients had functioning tumors that caused virilization in 98% of the girls and pre- cocious puberty in 93% of the boys. Bad prognosis was recorded in 18 patients (39.1%). Literature data indicate that relapse generally occurs within the first 18 months, and after this time there is a very good chance of cure9.29-32, similar to what has been observed in our own patients.

Different from reports in the literature, our study shows that girls are more affected than boys (2:1), probably because it is easier for families to notice the virilization of a girl as opposed to the signs of isosexual puberty in a boy. Despite the predomin- ance of females with AC, there was no difference between the sexes with regard to bad prognosis.

Another relevant fact in our study is the discrepancy in the histopathological findings and the follow up of the patients, confirming recent observations in the literature regarding the low prognostic value of this method, as well as PCNA analysis33,34. According to some authors, the nuclear content of the DNA may have a prognostic value, but there is a lot of controversy in recent studies regarding the use of this method to deter- mine the malignant potential11.35,36

A positive fact observed in this study is the higher incidence of bad outcome in patients with a

duration of disease less than or equal to 7 months, demonstrating that the greater the velocity of clinical events, the more aggressive the tumor. On the other hand, the older the child, the worse the prognosis. This finding calls attention to the possibility of late diagnosis in our patients. Another important observation was the presence of systemic hypertension as a marker of bad prognosis, even in children who did not present signs of Cushing’s syndrome, demonstrating a more prominent mineralo- corticoid activity, characterizing a mixed hormonal profile.

In 1987, Brennan37 observed that tumors located on the right side had more aggressive behavior, in accordance with our results. A possible explanation for this finding could be that the right-sided tumor is very close to the inferior vena cava and its venous drainage is made directly into it, facilitating tumoral dissemination.

Tumor size is an important prognostic marker, but it should be highlighted that some patients with a tumoral weight greater than 250 g had a benign course (patients # 3, 37 and 39), while only three patients (patients # 2, 4 and 15) with a tumoral weight less than or equal to 250 g presented a bad outcome. Several authors have also reported the importance of the weight or volume of the tumor as a prognostic marker 5,34,35,37,38

Study of the p53 protein, using a specific monoclonal antibody immunohistochemical method, allowed a retrospective study of paraffin-embedded tumoral material, giving a positive reactivity of approximately 54% when compared with the literature (between 19% and 27%)24,35,39. However, the most curious finding was the negative reactivity seen in cases with bad outcome, and positive reactivity in greater numbers among the cases with good prognosis. Perhaps the poor outcome in the negative cases is associated with very important gene alterations and production of extremely modified protein whose altered immunogenicity precludes its detection, as demonstrated by Reincke et al.39 in AC in adults via gene amplification.

The statistical analysis and mathematical model primarily enabled a prognostic evaluation based only on clinical data, as demonstrated by the J1 index, whose variables are directly related to the patient’s age, laterality of the tumor, presence of

JOURNAL OF PEDIATRIC ENDOCRINOLOGY & METABOLISM

TABLE 2 Follow up of adrenal cortex carcinoma
BadGoodTotalP
Age >33 mo16925
Age <33 mo21921<0.0009
Tumor side R10717
Tumor side L82129<0.036
Hypertension present131023
Hypertension absent51823<0.01
Length disease ≤7 mo141226
Length disease >7mo41620<0.01
Tumor weight ≤250 g32427
Tumor weight >250 g153180.000001
p53 negative9615
p53 positive513180.06
J1 ≤0.432023
J1>0.4158230.0002
J2 ≤0.402222
J2>0.4185230.0000001
J3 ≤0.431316
J3>0.4116170.007
J4 <0.501616
J4>0.5142160.000001
TABLE 3 Associated probabilities
Pr{B| J1 >0.4} = 64%Pr{B| J2 >0.4} = 76%
Pr{G| JI <0.4} = 84%Pr{G| J2 <0.4} = 96%
Pr{B| J3 >0.4} = 63%Pr{B| J4 >0.5} = 83%
Pr{G| J3 <0.4} = 78%Pr{G| J4 <0.5} = 94%

Pr = probability; B = bad; G = good; J1-4: indices.

systemic hypertension, and duration of disease. This index demonstrated a probability associated with a good outcome in 84% and a bad one in 64% of cases. When we associated the negative react- ivity of the p53 protein to this index, the probability of a good outcome was 78% and a bad one 63%. Adding the weight of the tumor to the J2 and J4 indices, we obtained the probabilities for a good outcome in 96% and 94%, and a poor outcome in 76% and 83%, respectively.

This study led us to conclude that the histo- pathological information does not correlate with the probable course and outcome of the disease. On the other hand, clinical data and tumoral weight are important as prognostic tools. The proposal of a simple mathematical model aims to offer a useful tool to deal with the prognosis of patients with AC.

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